CURRICULUM VITAE

CURRICULUM VITAE Name: Paola Leone Professional Address: Rowan University - School of Osteopathic Medicine 42 E. Laurel Road Stratford, NJ 08084 Phone# Education: website: http://www.rowan.edu/soinicgtweb/ University of Padua, Italy M.S-Ph.D. Neuroscience 1987 Citizenship: American, Italian Career: RESEARCH: 1988-1989 Ph.D. Research Internship Institute of Pharmacology and Experimental Toxicology, UNIVERSITY OF CAGLIARI, Cagliari, Italy 1990-1992 Postdoctoral Research Fellow Center for Studies in Behavioral Neurobiology, CONCORDIA UNIVERSITY, Montreal, Canada 1993-1995 Postdoctoral Research Fellow Section of Neurosurgery, Gene Therapy and Neurogenetics Laboratories YALE UNIVERSITY, New Haven, USA 1995-1998 Research Scientist Department of Neurosurgery, Gene Therapy and Neurogenetics Laboratories YALE UNIVERSITY, New Haven, USA 1997-1998 Visiting Scientist Gene Therapy Center Department of Molecular Medicine UNIVERSITY OF AUCKLAND, Auckland, New Zealand 1998-2001 Assistant Professor Department of Neurosurgery JEFFERSON MEDICAL COLLEGE, Philadelphia, PA, USA EFTA01110707 LEONE, Paola 1998-2001 Associate Director CNS Gene Therapy Center JEFFERSON MEDICAL COLLEGE, Philadelphia, PA, USA 2002-2007 Associate Professor Department of Surgery & Department of Molecular Genetics, Microbiology, and Immunology UMDNJ / Robert Wood Johnson Medical School Camden, NJ, USA 2002-2007 Adjunct Associate Professor Department of Neuroradiology, CHILDREN'S HOPSITAL OF PHILADELPHIA, Philadelphia, PA, USA 2007 to June 2013 Associate Professor Department of Cell Biology UMDNJ-School of Osteopathic Medicine Stratford, NJ, USA 2007 to June 2013 Director Cell & Gene Therapy Center UMDNJ-School of Osteopathic Medicine Stratford, NJ, USA July 2013 to date Associate Professor Department of Cell Biology ROWAN-School of Osteopathic Medicine Stratford, NJ, USA July 2013 to date Director Cell & Gene Therapy Center ROWAN-School of Osteopathic Medicine Stratford, NJ, USA 2006-2007 HOSPITAL APPOINTMENTS: Director of Surgical Research Department of Surgery Cooper University Hospital, Camden, NJ, USA 2 of 22 EFTA01110708 LEONE, Paola TEACHING: 1989 Instructor, Pharmacology, Concordia University, Montreal, Canada 1990 Instructor, Pharmacology, Concordia University, Montreal, Canada 1991 Instructor, Pharmacology, Concordia University, Montreal, Canada 1992 Instructor, Pharmacology, Concordia University, Montreal, Canada 2003 Instructor, Medical Physiology, RWJMS, NJ 2004 Instructor, Medical Physiology, RWJMS, NJ 2007 Lecturer: Pharmacology, UMDNJ/SOM, NJ 2007 Lecturer: Pharmacology, GSBS/SOM, NJ 2008 Lecturer: PBL: Topics in Cell Biology, GSBS/SOM, NJ 2008 Lecturer: Pharmacology, UMDNJ/SOM, NJ 2008 Lecturer: Pharmacology, GSBS/SOM, NJ 2009 Lecturer: Pharmacology, UMDNJ/SOM, NJ 2009 Lecturer: Pharmacology, GSBS/SOM, NJ 2010 Lecturer: PBL: Topics in Cell Biology, GSBS/SOM, NJ 2010 Lecturer: Pharmacology, UMDNJ/SOM, NJ 2010 Lecturer: Pharmacology, GSBS/SOM, NJ 2011 Lecturer: Pharmacology, UMDNJ/SOM, NJ 2011 Lecturer: Pharmacology, GSBS/SOM, NJ 2011 Course Director: Neuroscience, GSBS/SOM, NJ 2012 Lecturer: Pharmacology, UMDNJ/SOM, NJ 2012 Lecturer: Pharmacology, GSBS/SOM, NJ 2012 Lecturer: Human Genetics, UMDNJ/SOM, NJ 2012 Course Director: Neuroscience, GSBS/SOM, NJ 2013 Lecturer: Pharmacology, UMDNJ/SOM, NJ 2013 Lecturer: Pharmacology, GSBS/SOM, NJ 2013 Lecturer: Ethics of Clinical Trials, GSBS/SOM, NJ 2013 Lecturer: Human Genetics, UMDNJ/SOM, NJ 2013 Course Director: Epigenetics of Learning and Memory, GSBS/SOM, NJ PROFESSIONAL MEMBERSHIPS: American Society of Cell and Gene Therapy European Society of Cell and Gene Therapy The American Society of Human Genetics International Brain Research Organization Society for Neuroscience 3 of 22 EFTA01110709 LEONE, Paola HONORS/AWARDS: 1999 UNESCO Award, Career Achievement Award, Italy 2003 Canavan Research Illinois, Best Scientist of the Year Award, USA 2006 Woman of the Year, Lions Club, Italy 2009 Foundation of UMDNJ's Excellence in Research Award, USA 2010 Canavan Research Illinois, Scientific Achievement Award, USA 2011 International Rare Disease Day, Hall Of Fame 2012 International Rare Disease Day, Hall Of Fame 2012 Edward J. III Medical Foundation Award in Biomedical Research, USA 2013 International Rare Disease Day, Hall Of Fame 2013 American Academy of Neurology — NeuroFilm Festival 3m Prize COMMITTEE MEMBER 2002 Judging Committee for Resident's Research, Cooper Health System 2003 Research Oversight Committee, Cooper Health System, Camden, NJ 2003 Search Committee for Neurosurgery Associates and Chief, Cooper Health System, Camden, NJ 2003 Judging Committee for Resident's Research, Cooper Health System 2004 Judging Committee for Resident's Research, Cooper Health System 2005 Search Committee for the Director of the Coriell Institute, Camden, NJ 2005 Search Committee for Associate Research Dean, UMDNJ, Camden, NJ 2005 Reviewer, Duchenne Muscular Dystrophy Grants, MVP, Tucson, AZ 2005 Member, Research Oversight Committee, Cooper University Hospital, NJ 2005 Judging Committee for Resident's Research, Cooper Health System, NJ 2006 Chair of Data Safety Monitor Board, Gene Therapy for Duchenne Muscular Dystrophy Phase I Protocol, Columbus, OH 2007 NIH Study Section, Inborn Errors Of Metabolism, Bethesda, MD 2008 Executive Committee, UMDNJ-SOM, Stratford, NJ 2008 Recruitment Committee, UMDNJ-SOM, Stratford, NJ 2008 Research Committee, UMDNJ-SOM, Stratford, NJ 2009 1ST Committee, UMDNJ-SOM, Stratford, NJ 2009 Reviewer, Duchenne Muscular Dystrophy Grants, MVP, Tucson, AZ 2009 Scientific Committee / Reviewer, Gene Therapy for MPSIIIA, France 2009 Scientific Committee / Reviewer, Partnership For Cures, Chicago, IL 2009 Executive Committee, UMDNJ-SOM, Stratford, NJ 2010 Scientific Committee / Reviewer, Gene Therapy for MPSII IA, France 2010 Scientific Committee / Reviewer, Gene Therapy for MPSIIIA & MPSIIIB, France 2010-2014 NIH-Data Safety Monitoring Board, Gene Therapy for Batten Disease, New York, NY 4 of 22 EFTA01110710 LEONE, Paola SCIENTIFIC ADVISORY BOARD MEMBER National Tay Sachs and Allied Disorders, USA Late Onset Tay-Sachs Foundation, PA, USA Canavan Research. IL, USA Jacob's Cure, NY USA Canavan Research Foundation, CT Canavan Foundation, NY, NY Partnership for Cures, IL, USA Giant Axonal Neuropathy Foundation, NY National Organization of Rare Disease SERVICE ON MAJOR COMMITTEES: International Therapy for MPSIIIA, France, 2009 Gene Therapy for MPSIIIA, France, 2010 Gene Therapy for MPSIIIA & MPSIIIB, France, 2010 National Duchenne Muscular Dystrophy Grants, MVP, 2005 Search Committee for the Director of the Coriell Institute, 2005 Data Safety Monitor Board- Gene Therapy for Duchenne Muscular, 2006 Dystrophy Phase I Protocol NIH Study Section- Inborn Errors Of Metabolism, 2007 Duchenne Muscular Dystrophy Grants, 2009 Partnership For Cures, 2009 Medical School/University Search Committee for Associate Dean of Research - UMDNJ-RWJMS 2005 Research Committee, UMDNJ-SOM, 2008 Research Committee, UMDNJ-SOM, 2009 Research Committee, UMDNJ-SOM, 2010 Faculty Affairs Committee. UMDNJ-SOM2011 The American Osteopathic Association Commission on Osteopathic College Accreditation 2012 Faculty Affairs Committee, UMDNJ-SOM 2012 Faculty Affairs Committee, UMDNJ-SOM 2013 Department (Name, Inclusive Dates) 1ST Committee, UMDNJ-SOM Cell Biology, 2008 Executive Committee, UMDNJ-SOM Cell Biology, 2008 1ST Committee, UMDNJ-SOM Cell Biology, 2009 Executive Committee, UMDNJ-SOM Cell Biology, 2009 Research Committee, UMDNJ-SOM Cell Biology, 2010 1ST Committee, UMDNJ-SOM Cell Biology, 2010 Executive Committee, UMDNJ-SOM Cell Biology, 2010 1ST Committee, UMDNJ-SOM Cell Biology, 2011 Executive Committee, UMDNJ-SOM Cell Biology, 2011 5 of 22 EFTA01110711 LEONE, Paola 1ST Committee, UMDNJ-SOM Cell Biology, 2012 Executive Committee, UMDNJ-SOM Cell Biology, 2012 1ST Committee, UMDNJ-SOM Cell Biology, 2013 Executive Committee, UMDNJ-SOM Cell Biology, 2013 SERVICE ON HOSPITAL COMMITTEES: Judging Committee for Resident's Research, Cooper Health System, 2002 Research Oversight Committee, Cooper Health System, 2003 Search Committee for Neurosurgery Associates and Chief, Cooper Health System, 2003 Judging Committee for Resident's Research, Cooper Health System, 2003 Judging Committee for Resident's Research, Cooper Health System, 2004 Member, Research Oversight Committee, Cooper University Hospital, 2005 Judging Committee for Resident's Research, Cooper Health System, 2005 INVITED SPEAKER — SCIENTIFIC MEETINGS 2000 Hunter's Hope Conference, IL, USA 2001 Metabolic Disorder Conference, Netherland 2001 Late Onset Tay Sachs Conference, PA USA 2002 European Pediatric Conference, Italy 2002 Carlo Besta National Neurological Institute, Italy 2002 Milano Pediatria, Milano, Italy 2002 San Raffaele Research Institute, Italy 2002 Myelin Project Conference, FL, USA 2003 United Leukodystrophy Annual Conference, Chicago, IL, USA 2003 Workshop on Genetic Systems in the Rat, Kyoto, Japan 2003 Pelizaeus-Merzbacher Conference, VA, USA 2004 Center for Drug Evaluation, Taipei, Taiwan 2004 Jichi Medical School, Kawachi, Japan 2004 Myelin Project Annual Meeting, Aquiterme, Italy 2004 NAA Symposium, Bethesda, MD 2004 CNS Foundation, Tarrytown, NY 2005 National Tay Sachs & Allied Disorders, Annual Conference,New Orleans 2005 American Society of Gene Therapy. St Louis, MO 2005 Pharmaness Symposium, Pula, Italy 2006 Basic Science Seminar, UMDNJ, Newark 2006 Nuclean Medicine Conference, Cagliari, Italy 2006 Cell Biology Seminar, UMDNJ/SOM, Stratford, NJ 2006 Transgenic Rat Symposium, Tokyo, Japan 2007 Metachromatic Leukodystrophy Foundation, Gavoi, Italy 2008 European Leukodystrophy Association, Sevres, France 2008 Canavan Research Summit, White Plains, NY 2008 Annual Canavan Foundation Meeting, New York, New York 2009 Canavan Disease Conference, Tel Aviv, Israel 2010 Pediatric Leukodystrophy Workshop, Italy 2010 Columbia University-Medical Center, Neurology Grand Round, NY 2011 Canavan Research Summit, Harrison, NY 2012 The Translational Science of Rare Diseases, AAAS, Vienna, 2013 6 of 22 EFTA01110712 LEONE, Paola Grand Round-Neurology, Hackensack UMC, NJ 2013 European Leukodystrophy Association, Paris 2013 NIH Recombinant DNA Advisory Committee, Bethesda, MD 2013 United Leukodistrophy Foundation, IL INVITED SPEAKER - FUNDRAISING EVENTS 2002 Jacob's Cure — Fundraising Bowling Event, Strike Out Canavan Disease, NY 2002 Canavan Research Foundation — Tennis Tournament, a 2003 Jacob's Cure — Fundraising Bowling Event, Strike Out Canavan Disease, NY 2003 Canavan Research Illinois, Annual Event, IL 2003 Canavan Research Foundation — Golf Tournament, CT 2004 As Daylight Breaks — Documentary Release, NY 2005 Canavan Research Foundation - Golf Tournament, CT 2005 Jacob's Cure - Fundraising Bowling Event, Strike Out Canavan Disease, NY 2006 Women For Stem Cells Research - StemCellebration Foundation, NY 2007 Jacob's Cure - Lucky Magazine Event, LA 2009 Saving Lavi Life Fundraising Event — Tel Aviv, Israel 2010 Canavan Research Illinois, Annual Fundraising Event, IL 2010 Jacob's Cure, Fundraising Event, Dream Big Gala, NY 2011 Canavan Research Illinois, Annual Fundraising Event, IL 2012 Canavan Research Illinois, Annual Fundraising Event, IL CONFERENCE ORGANIZER & CHAIR 2000 Neurodegenerative Diseases Symposium, PA 2005 American Society of Gene Therapy, Symposium, St. Louis, MI 2008 Canavan Research Summit, White Plains, NY 2011 Canavan Research Summit, Harrison, NY Institutional Review Board Protocols: Institutions: Cooper Hospital (NJ), UMDNJ (NJ), Children's Hospital of Philadelphia (PA) Title: Gene Therapy of Canavan Disease using AAV-ASPA Role: Research P.I. at Cooper Hospital and UMDNJ; Co-PI at CHOP. Completed Institution: Cooper University Hospital (NJ), Title: Use of Lithium for treatment of patients with Canavan Disease Role: Research P.I. Completed Institution: Cooper University Hospital (NJ) Title: Use of Buspirone for treatment of patients with spinocerebellar ataxias Role: Co-Investigator Completed 7 of 22 EFTA01110713 LEONE, Paola Institution: Cooper University Hospital (NJ), Title: Warfarin for Metachromatic Leukodystrophy Role: Research P.I. Completed Institution: Rowan University - SOM Title: Neuroradiologic Markers of Disease Progression in Canavan Disease Role: Research P.I. Active Institution: Rowan University - SOM Title: Role of Invariant Natural Killer T Cells in Multiple Sclerosis Role: Research P.I. Pending Sponsor of FDA-Investigational New Drug (IND) Protocols: INDH 7303 (1998-2002) - Phase I: LPD-ASPA for Canavan Disease 'NIX/ 9119 (2001- 2009) - Phase 1/II: AAV-ASPA for Canavan Disease Pre-IND filed: Triheptanoin Therapy for Canavan Disease FUNDING HISTORY GRANT SUPPORT: Principal Investigator Funding Agency: UMDNJ Foundation Title: Stem Cell Therapy for Canavan Disease Project Dates: 04/01/13 — 03/31/14 Total Direct Costs: $25,000 Funding Agency: Canavan Research Illinois Title: Dietary Therapy for Canavan Disease Project Dates: 02/01/13 — 01/31/14 Total Direct Costs: $100,000 Funding Agency: Jacob's Cure Title: human Stem Cell Therapy for Canavan Disease Project Dates: 08/01/12 — 07/31/13 Total Direct Costs: $100,000 Funding Agency: NIH-NINDS Title: Murine Neural Stem Cells in an animal model of Canavan Disease Project Dates: 07/01/07 - 06/30/12 Direct Costs: $275,000 Funding Agency: NIH/NINDS Project number: ROl/NS42120 Title: Gene Therapy for Canavan Disease using AAV2-ASPA 8 of 22 EFTA01110714 LEONE, Paola Project Dates: 09/01/02 - 07/31/08 Total Direct Costs: $3,250,000 Funding Agency: Jacob's Cure, NY Title: "Characterization of an Animal Model of Canavan Disease" Project Dates: 07/01/07 - 06/30/09 Total Costs: 400,000 Funding Agency: ProEdoardo Foundation Title: Warfarin for Metachromatic Leukodystrophy Project Dates: 06/01/07-05/30108 Total Costs: $150,000 Funding Agency: Canavan Research Illinois & Jacob's Cure, NY Title: Analysis of Stem Cell Therapy in the Tremor Rat Model of Canavan Disease Project Dates: 06/01/03- 05/31/07 Total Direct Costs: $500,000 Funding Agency: Canavan Research Foundation, CT Title: Human Fetal Neural Stem Cell for Canavan Disease Project Dates: 06/01/06 - 05/31/07 Total Direct Costs: $50,000 Funding Agency: Canavan Research Illinois Title: Lithium Therapy for Canavan Disease Project Dates: 12/01/01- 07/31/04 Total Direct Costs: $200,000 Funding Agency: Canavan Fund, NY Title: Preclinical Studies for Gene Transfer of Canavan Disease Project Dates: 05/31/99 - 06/01/01 Total Direct Costs: $250,000 Funding Agency: Canavan Foundation, NY Type: Canavan Disease Fellowship Project Dates: 04/01/97 - 03/31/98 Total Direct Costs: $100,000 Funding Agency: Canavan Research Foundation, CT Title: Liposome-Based Vector Delivery for Canavan Disease Project Dates: 06/01/95 - 05/31/98 Total Direct Costs: $300,000 B, Co-Investigator NIH Center Grant on Epilepsy (1997-1998), Gene Therapy of Epilepsy, $2,700,000 NIH Program, Project Grant on Transmissible Dementias, (1995-2000), Etiology of Creutzfeldt—Jakob Disease, $1,700,000, Yale University, New Haven, CT 9 of 22 EFTA01110715 LEONE, Paola C. Pending 1) Phenotype and polyfunctionality of peripheral blood invariant Natural Killer T (iNKT) cells as an immunological marker for different forms of Multiple Sclerosis and following different immunomodulatory treatments. NMSS (P.I.) Direct Costs $579,387, Indirect Costs $20,502. 2) AAV2-ASPA-Based Metabolic Intervention for Alzheimer's Disease. NIH-SBIR R-43 (Co-Investigator), Direct Costs $150,000, Indirect Costs $84,000. 3) Triheptanoin for Canavan Disease. NIH- UO1, (P.I.) Direct Costs $350,000, Indirect Costs $206,500. 4) Regulation of Energy Homeostasis in Neurodegenerative Disease by N-acetylaspartate. NIH RFA-RO1 (co-Investigator), Direct Costs $800,000, Indirect Costs $ 472,000 BIBLIOGRAPHY: 1. Leone P., and Di Chiara G. (1987) Blockade of DI receptors by SCH 23390 antagonizes morphine- and amphetamine-induced place preference conditioning. Eur. J. Pharm., 135: 251-254. 2. Acquas, E., Carboni, E., Leone, P., and Di Chiara, G. (1988) 5HT3 receptor antagonists block morphine and nicotine- but not amphetamine-induced reward. Eur. J. Pharm., 97: 175-178. 3. Acquas, E., Carboni, E., Leone, P., and Di Chiara, G. (1989) SCH 23390 blocks drug- conditioned place preference and place-aversion: anhedonia (lack of reward) or apathy (lack of motivation) after dopamine-receptor blockade? Psychopharmacology, 99:151- 155. 4. Leone, P., Pocock, D. and Wise, R.A. (1991) Morphine-dopamine interaction: ventral tegmental morphine increases nucleus accumbens dopamine release as measured by in vivo microdialysis. Pharm Biochem. & Behay., 39: 469-472. 5. During, M.J., Mirchandani, G., Williamson, A., Leone, P., deLanerolle, N., Bergold, P.J., Federoff, H.J. (1993) Intrahippocampal injection of a HSV-1 vector expressing GluR6 induces spontaneous limbic seizures and CAI and CA3 hyperexcitability. Epilepsia, 34, (Suppl. 6): 75. 6. Devine, D.P., Leone, P., Pocock, D. and Wise, R.A. (1993) Differential Involvement of ventral tegmental Mu, Delta and Kappa opioid receptors in modulatoin of basal mesolimbic dopamine release: In vivo microdialysis studies J. Pharm. Exp. Ther., 266: 1236-1246. 7. Devine, D.P., Leone, P., Carlezon, A.W. Jr., and Wise, R.A. (1993) Ventral mesencephalic & opioid are involved in modulation of basal mesolimbic dopamine neurotransmission: an anatomical localization study. Brain Research, 622: 348-352. lOof 22 EFTA01110716 LEONE, Paola 8. Devine, D.P., Leone, P., and Wise, R.A. (1993) Striatal tissue preparation facilitates early sampling in microdialysis and reveals an index of neuronal damage. J. Neurochemistry, 61: 1246-1254. 9. Devine, D.P., Leone, P., and Wise, R.A.. (1993) Mesolimbic dopamine neurotransmission is increased by administration of µ-opioid receptor antagonists. Eur. J. Pharmarcology, 243: 55- 64. 10. During, M.J., Fried, I., Leone, P., Katz, A. and Spencer, D.D. (1994) Direct measurement of extracellular lactate in the human hippocampus during spontaneous seizures. J. Neurochem. , 62, 2356-2361. 11. Kaplitt, M.G., Leone, P., Xiao, X., Pfaff, D.W., O'Malley, K.L., Samulski, R.J., and During, M.J. (1994) Adeno-associated virus vectors yield long-term expression of potentially therapeutic genes in the mammalian brain. Nature Genetics, 8: 148-154. 12. During, M.J., Leone, P., Davis, K.E., Kerr, D., and Sherwin, R.S. (1995) Glucose availability influences hippocampal GABA release in the freely-moving rat via an effect on ATP-sensitive potassium channels. J. Clin. Inv., 95: 2403-2408. 13. Badiani, A., Leone, P., Noel, M.B., and Stewart J. (1995) Ventral tegmental area opioid mechanisms and modulation of ingestive behavior. Brain Research, 670: 264-276. 14. Maggs, D.G., Jacob, R., Langer, R., Leone, P., During M.J., Tamborlane, W.V., and Sherwin, R.S. (1995) Interstitial fluid concentrations of glycerol, glucose and amino acids in human quadricep muscle and adipose tissue: evidence for significant lipolysis in skeletal muscle. J. Clin. Inv., 96: 370-377. 15. Wise, P., Leone, P., Rivest, R., and Leeb, K. (1995) Elevations of nucleus accumbens dopamine and DOPAC levels during intravenous heroin self-administration. Synapse, 21, 140-148. 16. During, M.J. and Leone, P. (1995) Adeno-associated virus vectors for gene therapy for neurodegenerative disorders. Clinical Neuroscience, 3, 292-300. 17. Jacob R.J., Dziura J., Medwick, M.B., Leone, P., During, M.J., Shulman, G.I., and Sherwin, R.S. (1997) The effect of Leptin is enhanced by microinjection into the ventromedial hypothalamus. Diabetes 46 (1) 150-152. 18. Freese, A., Kaplitt, M.G., O'Connor, W.M., Abbey, M., Langer, D., Leone, P., During,M.J. (1997) Direct gene transfer into human epileptogenic hippocampal tissue with an adeno- associated virus vector - implications for a gene therapy approach to epilepsy. Epilepsia, 38: 759-766. nof 22 EFTA01110717 LEONE, Paola 19. During, M.J. and Leone, P. (1997) Targets for Gene Therapy of Parkinson's Disease: Growth Factors, signal transduction and promoters. Exp. Neurot, 144, 74-81. 20. O'Connor, W.M., Davidson, B.L., Kaplitt, M.G., Abbey, M.V., During, M.J., Leone, P., Langer, D., O'Connor, M.J., Freese, A. (1997) Adenovirus vector-mediated gene transfer into human epileptogenic brain slices: prospects for gene therapy in epilepsy. Exp. Neurol. 148, 167-78. 21. During, M.J., Samulski, R.J., Leone, P., Kaplitt, M.G., Freese, A. Xiao, X., Elsworth, J.D., Roth, R.H., Sladek, J.R.Jr., O'Malley, K.L. and Redmond, D.E. (1998) In vivo expression of therapeutic human genes for dopamine production in the caudates of MPTP-treated monkeys using an AAV vector. Gene Therapy 5, 820-827. 22. During, M.J., Xu, R., Young, D., Kaplitt, M.G., Sherwin, R.S., and Leone, P. (1998) Peroral gene therapy of lactose intolerance using an AAV vector. Nature Medicine, 4, 1131-1135. 23. Young, D.D., Lawlor, P., Leone, P., Dragunow, M., During, M.J. (1999) Environmental enrichment inhibits spontaneous neuronal apoptosis and prevents neuronal injury in the rat. Nature Medicine, 5, 448-453. 24. Leone, P., Janson, C.G., McPhee, S.J., During, M.J. (1999) Global CNS Gene Transfer for Childhood Neurogenetic Enzyme Deficiency: Canavan Disease. Current Opinions in Molecular Therapeutics, 1:4, 487-492 25. Janson, C.G., McPhee S.W.J., Leone, P., During, M.J. (1999) Gene Therapy in the Central Nervous System. Science and Medicine, 6, 4-5. 26. Leone, P., McPhee, S.W., Janson, C.G., Davidson, B.L., Freese, A., During, M.J. (2000) Multisite partitioned delivery of human tyrosine hydroxylase gene with phenotypic recovery in Parkinsonian rats. Neuroreport, 2:6, 1145-1151. 27. During, M.J., Symes, C.A., Lawlort, P.A., Lin, J., Dunning, J., Fitzsimons, H.L., Poulsen, D., Leone,P., Xu, R., Dicker, B.L., Lipski, J., Young, D. (2000) Targeted autoimmunity: a genetic NMDARI vaccine with efficacy in experimental stroke and epilepsy. Science, 287, 1453-1460. 28. Leone, P., Janson, C.G., Bilianiuk, L., Wang, Z., Huang, L., Sorgi, F., Matalon, R., Kaul, R., Zeng, Z., Freese, A., McPhee S.W., Mee, E., During, M.J. (2000) Aspartoacylase gene transfer to the mammalian central nervous system with therapeutic implication for Canavan Disease. Ann. of Neurol, 48:1, 27-38. 29. Telfeian, A.E., Federoff, H.J., Leone, P., During, M.J., Williamson, A. (2000) Overexpression of GluR6 in rat hippocampus produces seizures and spontaneous nonsynaptic bursting in vitro. Neurobiology of Disease, 4, 362-374. 30. Janson CG, McPhee SW, Leone P., Freese A, During MJ. (2001) Viral-based gene transfer to the mammalian CNS for functsional genomic studies. Trends Neurosci. 1;24(12):706-12. 12of 22 EFTA01110718 LEONE, Paola 31. Xu R, Janson CG, Mastakov M, Lawlor P, Young D, Mouravlev A, Fitzsimons H, Choi KL, Ma H, Dragunow M, Leone P., Chen Q, Dicker B, During MJ. (2001) Quantitative comparison of expression with adeno-associated virus (AAV-2) brain-specific gene cassettes. Gene Therapy 8 (17):1323-32. 32. Janson CG, Ramesh T.M., During, M.J., Leone, P., Heywood, J.(2001) Human intrathecal transplantation of peripheral blood stem cells in amyotrophic lateral sclerosis. Hematotherapy & Stern Cell Research 10:6, 913-16. 33. Abi-Saab W.M., Maggs D.G., Jones T, Jacob R, Srihari V., Thompson J., Kerr D, Leone P, Krystal J.H., Spencer D.D., During MJ, Sherwin R.S. (2002) Striking Differences in Glucose and Lactate Levels Between Brain Extracellular Fluid and Plasma in Conscious Human Subjects: Effects of Hyperglycemia and Hypoglycemia. J Cereb Blood Flow Metab. (3): 271-279. 34. Janson, C.G, McPhee, S.W.J., Bilaniuk, L., Haselgrove, J., Testaiuti, M., Freese, A., Wang, D.J., Shera, D., Hurh P., Rupin J., Saslow, E., Goldfarb, O., Goldberg, M., Larijani, G., Sharrar, W., Camp A., Liouterman, L., Kolodny, E., Samulski, J., Leone, P. (2002) Gene Therapy of Canavan Disease: AAV-2 Vector for Neurological Delivery of Aspartoacylase Gene (ASPA) to the Human Brain. Human Gene Therapy, 13: 1391-1412. 35. Janson C.G., Leone P., Freese, A. (2002) Current medical and surgical approaches to Parkinson's Disease. Science and Medicine. 8(6), 328-338. 36. Janson C.G., Leone P., Freese, A. (2003) Current medical and surgical approaches to Parkinson's Disease. Science & Medicine 9(1): 24-35. 37. Janson C.G., Leone P., Freese, A. (2003) Current medical and surgical approaches to Parkinson's Disease. Science & Medicine 9(2): 82-95. 38. Zeng, B., Wang, Z-H., Ribeiro, L.A., Leone, P., De Gasperi, R., Kim, S-J., Raghavan, S., Ong, E., Naum, M., Pastores, G.M., Kolodny, E.H. (2003) Identification and Characterization of Novel Mutations of the Aspartoacylase Gene in Non-Jewish Patients with Canavan Disease. Journal of Inherited Metabolic Diseases. 2002; 25: 557-570. 39. Janson C.G., During M., Shifman Y, Leone P. (2003) Transcriptional Activation of HumanCREB gene promoter using bis-PNA (peptide nucleic acid). Letters in Peptide Science. 10 (3), 373-376. 40. Boulis, N.M., Noordmans, A.J., Song, D.K., Imperiale, M.J., Rubin, A., Leone, P., During, M., Feldman, E. (2003) Adeno-Associated Viral Vector Gene Expression in the Adult Rat Spinal Cord Following Remote Vector Delivery. Neurobiology of Disease. 14(3): 535-41 41. McPhee, S.W.J., Francis, J.S., Janson, C.G., Serikawa, T., Hyland, K., Ong, E.O., Raghavan, S.S., Freese, A., Leone, P. (2004) Effects of AAV-2 mediated aspartoacylase gene transfer in the tremor rat model of Canavan disease. Molecular Brain Research. 135(1- 2):112-21 i3of 22 EFTA01110719 LEONE, Poole: 42. Inoue, H., Osawa, I., Murakami, T., Kimura, A., Hakamata Y., Sato, Y., ICaneko,T., Okada, T., Ozawa, K., Francis J., Leone' P., and Kobayashi, E. (2005) Development of Inbred Transgenic Strains of Rats with LacZ or GFP. Biochemical & Biophysical Research Communication. 329(1): 288-95. 43. Assadi, M., Janson, C.G., Bilaniuk, L., Shera D., Leone, P. (2005) Case Report: Lithium Citrate for Canavan Disease. Pediatric Neurology. 33:235-243. 44. Tavazzi, B., Lazzarino, G., Amorini, A.M., Bellia, F., Fazzina, G., Giardinia, B., Leone, P. (2005) Sensitive and reproducible high performance liquid chromatographic separation of N- Acetyl Aspartate, N-Acetyl Glutamate and N-Acetyl-Aspartyl Glutamate suitable for the biochemical evaluation of Canavan Disease patients. Clinical Biochemistry. 38(11):997-1008. 45. Janson, C.G., Kolodny, E.H., Bilaniuk, L., Shera, D., Goldfarb, O., Zeng, B-J., Leone, P. (2006) Mild-onset presentation of Canavan disease associated with novel G212A point mutation in aspartoacylase gene. Annals of Neurology. 59(2):428-31. 46. McPhee, S.W.J., Janson, C.G., Francis, J.S., Samulski, R.J., Freese, A., Shera, D., Leone, P. (2006) Immunological Profile of Patients Treated With AAV-2 Vectors. Journal of Gene Medicine. 8(5):577-88 47. Francis, J.S., McPhee, S.W.J., Olariu, A., Janson, C.G., Leone, P. (2006) A Role for Aspartoacylase in the Regulation of BDNF and the Timing of Postnatal Oligodendrogenesis. Journal of Neuroscience Research. 84(1): 151-69. 48. Zeng, B-J., Wang, Z-H; Torres, P.A., Pastores, G., Leone, P. Raghavan, S.S., Kolodny E.H. (2006) Rapid Detection of Three Large Novel Deletions of the Aspartoacylase Gene In Non- Jewish Patients With Canavan Disease. Molecular Genetics and Metabolism. 89(1-2):156-63. 49. Zeng, B.J., Pastores G.M., Leone, P., Raghavan, S., Wang Z.H., Ribeiro, L.A., Torres, P., Ong E., Kolodny, E.H. (2006) Mutation Analysis of the Aspartoacylase Gene in non-Jewish Patients in Canavan Disease. Advances in Experimental Medicine and Biology. 576:165-73. 50. Janson CG, McPhee SWJ, Francis J, Shera D, Assadi M, Hurh P, Haselgrove J, Wang DJ, Bilaniuk L, Freese A, Leone P. (2006) Natural History of Canavan Disease Revealed by Serial MRI and Proton magnetic resonance spectroscopy (1H-MRS). Neuropediatrics. 84(1): 151-69. 51. Francis, J.S., Olaris, A., McPhee, S.W.J., Kobayashi, E, Leone, P. (2007) GFP-transgenic Lewis rats as a cell source for oligodendrocyte replacement. Journal of Experimental Neurology. 205(1):177-89. 52. Aghai, Z.H., Kode, A., Saslow J.G., Nakhla T., Farhath, S., Stahl, G.E., Eydelman R., Louise Strande, L., Leone, P., Rahman, I. (2007) Azithromycin Suppresses Activation of Nuclear Factor-kappaB and Synthesis of Pro-inflammatory Cytokines in Tracheal Aspirate Cells from Premature Infant. Pediatric Research. 62(4):483-8. 14of 22 EFTA01110720 LEONE, Paola 53. Aghai, Z. H., Faqiri, S., Saslow, J.G., Nakhla, T., Farhath, S., Kumar, A., Eydelman, R., Strande, L., Stahl, G.E., Leone P., and Bhandari. V. (2008) Angiopoietin 2 is Increased in Tracheal Aspirates From Premature Infants Developing Bronchopulmonary Dysplasia. Journal of Perinatology. 28(2):149-55. 54. Assadi, M., Basemen S., Janson C.J., Wang D-J., Bilaniuk L., Leone P. (2008) Serial 1HMRS in GM2 Gangliosidoses. European Journal of Pediatrics,. 167 (3):347-52. 55. Assadi, M., Campellone, J.V., Janson C.G., Veloski, J.J., Schwartzman R.J., Leone, P. (2008) Treatment of Spinocerebellar Ataxia with Buspirone . Journal of Neurological Sciences. 260(1- 2):143-146. 56. Assadi, M., Leone, P., Veloski J.J., Schwartzman R.J. Janson C.G., Campellone J.V.(2008) Validating an Ataxia Functional Composite Scale in Spinocerebellar Ataxia. Journal of Neurological Science 268(1-2): 136-9. 57. Wang J., Leone P., Wu G., Francis J.S., Li H., Jain M.D., Serikawa t., Ledeen R.W. (2009) Myelin Lipid Abnormalities in the Aspartoacylase-Deficient Tremor Rat. Neurochemical Research. 34(1) :13848. 58. Assadi M, Janson C, Wang DJ, Goldfarb O, Suri N, Bilaniuk L, Leone P. (2010) Lithium citrate reduces excessive intra-cerebral N-acetyl aspartate in Canavan disease. Eur J Paediatr Neurol. 4(4):354-9. 59. Assadi M., Wang D-J, Andserson, K., Carran M., Bilaniuk, L., Leone, P. (2012) Vitamin k antagonist warfarin for palliative treatment of metachromatic leukodystrophy, a compassionate study of four subjects. J. of Cent. Nerv. Syst. Dis.. (4) 73-79. 60. Francis, JS, Leone P. (2012) Developmental Regulation of aspartoacylase expression by glutamatergic activity in vivo. Glia.59(10):1435-46 61. Leone, P., McPhee SWJ, Shera D., Samulski R.J., Kolodny E., Bilaniuk L., Wang D-J, Goldfarb O., Assadi M., Goldman W., Freese A., Janson C.G., (2012) Clinical trial of AAV2-ASPA gene therapy for Canavan disease: long-term results of recombinant adeno- associated viral vector in the human brain. Sci. Trans. Med.19;4 (165) 1-13. 62. Assadi M., Wang D-J, Velazquez-Rodriquez, Leone P. (2013) Multi-voxel IH-MRS in Metachromatic Leukodystrophy. J. Cent. Nerv. Syst. Dis. Apr 26;4:73-9. 63. Janson, C., Romanova L.G., Leone P., Nan Z., Belur L., Mclvor S.R., Low W. Comparison of Endovascular and intraventricular gene therapy with AAV5-IDUA for Hurler's Disease. (2013) Neurosurg. In press 64. Francis J.S, Markov V, Leone P. (2013) Dietary Triheptanoin Rescues White Matter Degeneration and Motor Function in the Nur7 Mouse Model of Canavan Disease. J Inherited Met Dis (submitted) 15of 22 EFTA01110721 LEONE, Paola Books: Editor: Janson CG, Leone P, Simeone F, Freese AM, Eds. Molecular Neurosurgery (Progress in Neurosurgery, Volume #17), Karger, Basel, 2005 Abstracts 1992 — current 1. Leone, P., Rivest, R., and Wise, R.A. (1992) Microdialysis assessment of nucleus accumbens dopamine and metabolites during intravenous heroin self-administration and extinction. Society for Neuroscience, 18: 157.10. 2. Devine, D.P., Leone, P., and Wise, R.A. (1992) Extracellular nucleus accumbens dopamine is increased by microinjections of selective m opioid antagonists into the ventral tegmental area. Society for Neuroscience, 18: 417.6. 3. Leone, P., Devine, D.P., Pocock, D., and Wise, R.A. (1992) Modulation of mesolimbic dopamine by ventral tegmental area p, 8, and k opioid receptors: in vivo microdialysis studies. Dopamine 92, Italy. 4. During, M.J., Mirchandani, G., Williamson, A., Leone, P., de Lanerolle, N., Bergold, P.J., Federoff, H.J. (1993) Intrahippocampal injection of a HSV-1 vector expressing GluR6 induces spontaneous limbic seizures and CAI and CA3 hyperexcitability. Epilepsia, 34 : 75. 5. Leone, P., Geller, A.I., During, M.J. (1993) A HSV-1 vector expressing unregulated PKC from the TH promoter improves spatial navigation performance following intrahippocampal injection in young adult rats. Journal of Neurochemisty, 61: 135. 6. Leone, P., Dragunow, M., Davis, K.E., Ullrey, D., O'Malley, K.L., Neve, R., Geller, A.I., During, M.J. (1993) Hippocampal injection of a HSV-1 vector expressing an unregulated PKC from the TH promoter increases NE release, induces KROX24 expression in dentate granule cells and improves spatial navigation performance in rats. Society for Neuroscience, 19: 328.1. 7. Mirchandani, G.R., Leone, P., Collins, W.F., During, M.J. (1993) Improved spatial learning by perfusion of NMDA in rat CAI hippocampus. Society for Neuroscience, 19: 325.12. 8. Fried, I., Leone, P., Kaiser, M.G., Puce, I.A., Nobre, A.C., Spencer, D.D., McCarthy, G., During, M.J. (1993) A microdialysis study of the effects of cognitive processing on norepinephrine release in the human hippocampus. Society for Neuroscience , 19: 173.8. 9. During, M.J., Mirchandani, G.R., Leone, P., Williamson, A., de Lanerolle, N.C., Geschwind, M.D., Bergold, P.J., Federoff. (1993) Direct hippocampal injection of a HSV-1 vector expressing GLUR6 results in the spontanous seizures, hyperexcitability in CAI cells, hilar and CA3 neurons. Society for Neuroscience, 19: 16.4. 10. Song, S., Leone, P., Wang, Y., Hartley, D., Bryan, J., Ullrey, D., Bak, S., Davis, K., Haycock, K., O'Malley, K., Neve, R., Geller, A., During, M. (1993) Rotational behavior in adult rats is induced by selective activation of the protein kinase C pathway in substantia nigra pars 16of 22 EFTA01110722 LEONE, Paola compacta neurons with a HSV-I vector. Society for Neuroscience, 19: 328.2. 11. Rivest, R., Leeb, K., Leone, P., Wise, R.A. (1993) Microdialysis assessment of nucleus dopamine and metabolites during intravenous self administration; changes with unit dose. Society for Neuroscience, 19: 417.14. 12. During, M.J., Mirchandani, G., Leone, P., Williamson, A., de Lanerolle, N.C., Geschwind, M.D., Federoff, H.J. (1993) Intrahippocampal injection of a HSV-I vector expressing GLUR6 induces spontanous limbic seizures and CAI and CA3 hyperexcitability. American Epilepsy Society. Baltimore, MD. 13. Wise, R.A., Leone, P., Rivest, R.,Leeb, K. (1993) Elevation of nucleus accumbens dopamine, and metabolite levels during IV heroin self-administration: independence of unit dose and response requirement. College of problems of Drug Dependence, 55 Annual Scientific Meeting, Toronto, Canada, 14. Leone,P., and During, M.J. (1993) A HSV-I vector expressing unregulated PKC from the TH promotor improves spatial navigation performance following intrahippocampal injection in young adult rats. l4th ISN meeting, Montpellier, France. 15. Devine, D.P., Leone, P., Wise, R.A. (1993) Surgical preparation of striatal tissue facilitates early sampling in microdialysis and reveals an index of neuronal damage. Third International Symposium on Microdialysis and Allied Analytical Techniques, Current Separations, 12: 48. Montreal, Canada 16. Leone, P., During, M.J. (1993) Modulation of norepinephrine release by hippocampal injection of a PKC expressing HSV-1 vector influences spatial navigation performance in the adult rat. Third International Symposium on Microdialysis and Allied Analytical Techniques, Current Separations, 12: 79. Montreal, Canada 17. During, M.J., Kaplitt, M.G., Leone, P., Davidson, B., Xiao, X., Samulski, R.J., Elsworth, J.D., Roth, R.H., Sladek, J.R., Redmond, D.E., O'Malley, K.L. (1994) AAV and adenovirus mediated transfer of human tyrosine hydroxylase into rat and primate brain. Cold Spring Harbor Meeting on Gene Therapy, abstr. 190. 18. Samulski, R., Xiao, X., Kaplitt, M., Leone, P., Pfaff, D., O'Malley, K., During, M. (1994) Generation of AAV vectors for transduction and expression in neuronal cells in vivo. Cold Spring Harbor Meeting on Gene Therapy, abstr. 9. 19. Leone, P., Sherwin, R.S., Davis, K.E., Wallace, E., Dragunow, M., During, M.J.(1994) Cognitive improvement and ICROX24 induction in the lateral amydgala after D-glucose administration. Society for Neuroscience, 20: 334.23. 20. Williamson, A, Telfeian, A.E., Leone, P., Federoff, H.J., During, M.J.(1994) Overexpression of GI R6 in rat hippocampi produces spontaneous non-synaptic bursting in vitro. Society for Neuroscience, 20: 680.3. 21. Kaplitt, M.G., Leone, P., Freese, A., Xiao, X., Pfaff, D.W., O'Malley, K.L., During, M.J.( 1994) Adeno-associated virus (AAV) vectors yield safe delivery and long-term expression of potentially therapeutic genes in the adult mammalian brain. Society for Neuroscience, 20: 602.11. 22. During, M.J., Kaplitt, M.G., Leone, P., Xiao, X., Elsworth, J.D., Roth, R.H., Sladek, J.R., Redmond, D.E. (1994) AAV mediated transfer of tyrosine hydroxylase and aromatic amino acid 17of 22 EFTA01110723 LEONE, Paola decarboxylase genes into the primate brain: A direct gene therapy approach to Parkinson's Disease. Society for Neuroscience, 20: 602.10. 23. Kaplitt, M.G, Rosenfeld, M.R., Pfaff, D.W., Meneses, P., O'Malley, K.L., Leone, P., During, M.J.(1994) Adeno-Associated Virus (AAV) Vectors for Genetic Therapy of Neurological Disease. American Association of Neurology. 24. Leone, P. and M.J. During (1994) Hippocampal norepinephrine: from genetic intervention in the rat to cognition in man. 4thSpring Hippocampal Research Conference, Grand Cayman, BWI. 25. During, M.J., Kaplitt, M.G., Leone, P., Freese, A., Samulski, R.J., Elsworth, J.D., Roth, R.H., Sladek, J.R., Redmond, D.E., O'Malley, K.L. (1995) In vivo gene therapy of Parkinson's Disease in a primate model. Amer. Assoc. Neurol. Surg. 62: 702. 26. Williamson, A., Telfeian, A., Mirchandani, G., Leone, P., Federoff, H.J., During, M.J. (1995) Viral vector mediated transduction of hippocampal neurons with GluR6 in vivo: A genetic model of acquired synaptic excitability. Neurobiology of Epilepsy Workshop. 27. Redmond, D.E., O'Malley, K.L., Kaplitt, M.G., Samulski, R.J., Elsworth, J.D., Sladek, J.R., Leone, P., Xiao, X., Freese, A., Taylor, J.R., Roth, R.H., During, M.J. (1995) AAV vectored gene therapy in primate parkinson's model. Society for Neuroscience, 21: 225.9 28. During, M.J., Choi-Lundberg, D., Leone, P., Lin, Q., Kaplitt, M.G., Xiao, X., Samulski,R.J., Freese, A., Hoffer, P., Roth, R.H., Elsworth, J.D., Sladek, J.R., O'Malley, K.L., Federoff, H.J., Redmond, D.E., Bohn, M.C. (1995) Neuroprotection and Neuroregeneration in rodent and primate parkinsonian models using AAV vectors expressing GDNF. Society for Neuroscience, 21: 224.22 29. Leone, P., and During, M.J. (1995) Selective involvement of hippocampal norepinephrine in musical perception and acquisition-recognition task in the human brain: a microdialysis study. Society for Neuroscience, 21: 303.8. 30. During, M.J., Choi-Lundberg, D., Leone, P., Lin, Q., Kaplitt, M.G., Xiao, X., Samulski, R.J., Freese, A., Hoffer, P., Roth, R.H., Elsworth, J.D., Sladek, J.R., O'Malley, K.L., Federoff. H.J., Redmond, D.E., Bohn, M.C. (1995) Neuroprotection and neurodegeneration in rodent and primate parkinsonian model using AAV vectors expressing GDNF. Society for Neuroscience, 21: 303.8. 31. Williamson, A., Telfeian, A., Mirchandani,G., Leone, P., Federoff, H.J., During, M.J.(1995) Viral vector mediated transduction of hippocampal neurons with GluR6 in vivo : A genetic model of acquired synaptic excitability. Neurobiology of Epilepsy Workshop. 32. Samulski, R., Xiao, X., Kaplitt, M., Leone, P., Pfaff, D., O'Malley,K., During, M.J. (1995) Generation of AAV vectors for transduction and expression in neuronal cells in vivo. Cold Spring Harbor Meeting on Gene Therapy, 9. Society for Neuroscience 33. During, M.J., Kaplitt, M.G., Leone, P., Davidson, B., Xiao, X., Samulski, R.J., Elsworth, J.D., Roth, R.H., Sladek, J.R., Redmond, D.E., O'Malley, K.L.( 1995) AAV and adenovirus mediated transfer of human tyrosine hydroxylase into rat and primate brain. Cold Spring Harbor meeting on Gene Therapy, 190. 34. During, M.J., Zeng, Z., Kaul, R., Sorgi, F., Huang, L., Mee, E., Wang, J., Mayes, L., Ment, L., Leone, P. (1996) Direct CNS gene transfer into the human brain using a liposome- polymer-AAV plasmid vector. Cold Spring Harbor Meeting on Gene Therapy. 35. During, M., Zeng, Z., Kaul, E., Sorgi, F., Huang, L., Mee, E., Wang, J., Mayes, L., Ment, L., Leone, P. (1996). Gene Therapy of Canavan Disease. Society for Neuroscience, 22: 18.12. 18of 22 EFTA01110724 LEONE, Paola 36. Leone, P., Young, D., Sorgi, F.L., Huang, L., Young, D.D., Fan,X., Xie, H., Sherwin, R., and During, M.J. (1997) Programmed gene expression in retrogradely targeted areas of the CNS by viral and non-viral vectors. Society for Neuroscience, 23: 319.8 37. During, M.J., Nass, D.J., Xie, H., Freese, A., Leone, P. (1997) Towards gene therapy of epilepsy: comparision of GAD-65, GABA-A-Delta subunit and GDNF vectors in rat kainate. Society for Neuroscience, 23: 119.5 38. Freese, A.,Young, D.D., Liu, C.Y., Leone, P., Davidson, B.L., Silver, D., Xie, H., During, M.J. (1997) Phenotypic recovery in Parkinsonian rats: relationship between numbers of transduced cells and behavior using an adenoviral vector expressing tyrosine hydroxylase. Society for Neuroscience, 23: 211.3 39. During, M.J., Young, D.D., Liu, C.Y., Davidson, B.L., Leone, P. (1998) Complete phenotypic recovery in Parkinsonian rats: is gene transfer of tyrosine hydroxylase alone suficient and what is the relationship between transduction efficiency and behavior. American Society of Gene Therapy, 402 40. Leone, P., During, M.J. (1998) Towards gene therapy of epilepsy: comparisons of GAD- 65, GABA-A delta subunit and GDNF vectors in a rodent model of temporal lobe epilepsy. American Society of Gene Therapy, 406. 41. During, M.J., Duncan, C., Sorgi, F., Mee, E., Huang, L., Bilaniuk, L.T., Wang, Zyuc., Seashore, M.R., Leone, P. (1998) Direct gene transfer into the human brain for the neurogenetic disorder, Canavan Disease, American Society of Gene Therapy, 458 42. Leone, P., Duncan, C., Barolat, G., Freese, A., Searshore, M., Sorge, F., Cherniske, E., Janson, C.G., During, M.J. (1998) Gene therapy of Canavan disease using LPD/pAAV-ASPA: a phase I study. Society for Neuroscience, 24: 405.4 43. Young, D., Symes, C., Lawlor, P., Fitzsimons, H., Ma, H., Xu, R., Leone, P., During M.J. (1999) Peroral gene transfer using rAAV Vectors. American Society of Gene Therapy, 191 44. McPhee, S.J., Fenu, S., Xu, R., Mastakov, M., Fan, X., Freese, A., During, M.J., Leone, P. (1999) In vivo analysis of tyrosine hydroxylase expression cassettes in the 6-OHDA Parkinsonian rat model using AAV vectors: a repeated injection paradigm. American Society of Gene Therapy, 254. 45. Leone, P., Freese, A., Latsko, M., McPhee, S., During, M.J., Janson, C.G., (2001) Comparison of viral & non-viral vectors for gene therapy of canavan disease. European Metabolic Group Meeting, Amsterdam. 46. McPhee, S.W.J., Horst, R., Janson, C.G., Serikawa, T., Jiao, X.Y., Young, D., During, M.J., Kolodny, E., Hyland, K., Leone, P. (2001) Towards phenotypic correction in a leukodystrophy model using AAV gene transfer to the CNS. Society for Neuroscience, 27. 47. L. Liouterman, During, M.J., McPhee, S.W.J., Freese, A., Janson, C.G., Horst, R., Peschle, C., Valtieri, M., Leone, P., (2001) Transplantation of human hematopoetic stem cells into rat brain: Analysis of cell survival and differentiation. Society for Neuroscience, 27. 48. Leone, P., Latsko, M., Haselgrove, J., McPhee, S.W.J., Janson, C.G., Jiao, X.Y., Liouterman, L., Francis, J., Young, D., Lawlor, P.A., Fitzsimons, H., Raniga, S., Bilaniuk, L., Wang, Z., Grant, M., Liu, Y., During, M.J., Freese, A., (2001) Gene therapy of canavan disease using AAV vectors: A Phase I clinical trial. Society for Neuroscience, 27 19of 22 EFTA01110725 LEONE, Pooke 49. Janson, C.G., McPhee, S.W.J., Leone, P. (2002) The first Phase I clinical trial using AAV in the human brain: AAV-ASPA for treatment of canavan disease. European Society of Gene Therapy, Antibes, France 50. Leone, P., McPhee, S.W.J., Janson, C.G., Liouterman, L., Freese A.(2002) Brain Gene Transfer using AAV vectors in the Canavan (Tremor) Rat Model and in children affected by Canavan Disease: Targeted Development of Preclinical and Clinical Studies. XIVth International Workshop on Genetic Systems in the Rat, Kyoto, Japan. 51. Leone P., McPhee, S.W.J., Janson,C.G., Freese A., Samulski J., Haselgrove J., Wang D.J., Bilaniuk L. (2002) Terapia genica nei pazienti con malattia metabolica: it modello della malattia di Canavan, Milanopediatria, Milano, Italy. 52. Leone P., McPhee, S.W.J., Janson,C.G., Samulski J., Wang D.J., Bilaniuk L. (2003) Clinical Trials in Gene Therapy Research. Gene Therapy Symposium, Taipei, Taiwan. 53. Leone P., McPhee, S.W.J., Janson,C.G., Samulski J., Wang D.J., Bilaniuk L. (2003) AAV- ASPA for Canavan Disease. Jichi Medical School, Kawachi, Japan. 54. Leone P., McPhee, S.W.J., Janson, C.G. (2004) Preliminary results of the Gene Therapy Phase I study for Canavan Disease. NAA Symposium, Bethesda, M.D. 55. Leone P., (2004) Stem Cells Therapies for White Matter Diseases. CNS Foundation Symposium, Tarrytown, NY. 56. Leone, P., McPhee, S.W.J., Janson,C.G., Samulski J., Wang D.J., Bilaniuk L. (2004) Gene Therapy for Canavan Disease. Myelin Project Annual Meeting, Aquiterme, Italy. 57. Leone., P. McPhee, S.W.J., Janson,C.G., Samulski J., Wang D.J., Bilaniuk L. (2005) Gene Therapy Case Study Report. National Tay Sachs & Allied Disorders, New Orleans, LA. 58. Leone, P., McPhee, S.W.J., Janson,C.G., Samulski J., Goldfarb, O., Saslow, E., Goldman, H.W., Wang D.J., Bilaniuk L. (2005). Safety and Efficacy of AAV-mediated Gene Transfer for Canavan Disease. American Society of Gene Therapy. Symposium - Clinical Trials: Metabolic Disorders, St Louis, MI. 59. Leone, P., McPhee, S.W.J., Janson,C.G., Samulski J., Goldfarb, O., Saslow, E., Goldman, H.W., Wang D.J., Bilaniuk L. (2005). Cell and Gene Based Experimental Therapies for White Matter Diseases. Pharmaness Symposium, Pula, Italy. 60. Olariu, A., Francis, J.S., McPhee S.W.J., Kobayashi, E., Leone, P. (2006) Neural Stem Cell Therapy for Canavan Disease. American Society of Gene Therapy, Baltimore, MD. Molecular Therapy, Vol. 13, S- 402. 61. McPhee, S.W.J., Janson, C.G., Li., C., Samulski, R.J., Camp, A.S., Francis, J.S., Shera, D., Freese, A., Aita, A.L., Goldman, H.W., Leone, P. (2006) The Systemic Human Immune Response to Intraparenchymal Administration of AAV. .American Society of Gene Therapy, Baltimore, MD. 62. Aghai ZH, Faqiri S, Saslow J, Nakhla T, Kumar A, Eydelman R, Strande L, Stahl G, Leone P, Bhandari V. (2007) Angiopoietin 2 is Increased in Tracheal Aspirates From Premature Infants Developing Bronchopulmonary Dysplasia. PAS 2007;61:5555.8. Platform Presentation. Eastern Society of Pediatric Research Annual Meeting, Philadelphia. 20of 22 EFTA01110726 LEONE, Paola 63. Aghai ZH, Faqiri S, Nakhla T, Saslow J, Kumar A, Eydelman R, Strande L, Stahl G, Leone P, Bhandari V. Dexamethasone Suppresses Angiopoietin 2 in Tracheal Aspirates of Ventilated Premature Infants. PAS 2007:5893.341. (2007) Platform Presentation, Eastern Society of Pediatric Research Annual Meeting, Philadelphia. 64. Moeller, M., DeFilippis, L., Vescovi, A., Francis, J.S., Leone, P. A Multi-Step Strategy for Maximizing Oligodendrocyte Progenitor Cell Yield From Human Neural Stem Cells. (2008) International Society for Stem Cell Research, Annual Meeting, Philadelphia. 65. Francis, J.S. & Leone, P. Aspartoacylase and Postnatal Oligodendrogenesis (2008). Canavan Research Summit, White Plain, NY 66. Snepar R., Francis JS., Strande L., Leone P. (2009) The Effects of NAA on Stem Cell Derived Oligodendrocytes In Vitro. 13'h Annual Stratford Research Day, Stratford. 67. Desai R., Bilaniuk L., Wang D.J.' Anderson-Thomas K.' Leone, P. Assessing Progression of Pediatric Leukodystrophies with 114 MRS & DTI. (2010)14'" Annual Stratford Research Day, Stratford. 68. Kira Moore, Francis., J.S. Leone, P. (2011) N-acetylaspartate catabolism is neuroprotective against oxidative stress. 15'h Annual Stratford Research Day, Stratford. 69. Francis J.S., Strande L., Markov V., Leone P. (2011) Oxidative stress precedes pathology in the Nur7 mouse model of Canavan's Disease. Canavan Research Summit 2011, Harrison, NY 70. Leone P, Vladimir M., Francis J., Therapeutic Potential of Human Embryonic Stem Cell- Derived Oligodendrocyte Progenitors for Canavan Disease. (2012). Philadelphia, 283, ASGT 71. Leone, P. Back to the Future: The Evolution of Translational Therapies for Canavan Disease. (2012) Vienna, Days of Molecular Medicine, AAAS 72. Leone, P. Translational Therapies for Canavan Diseases (2013), Paris, European. Leukodystrophy Association 73. Leone P, Vladimir M., Francis J., Therapeutic Potential of Human Embryonic Stem Cell- Derived Oligodendrocyte Progenitors for Canavan Disease. (2013) ) Stratford, NJ, UMDNJ/SOM Research Day. 74. Manzoor A., Francis J., Markov V., Shah Z., Leone P. Triheptanoin Treatment in the Nur7 Mouse Model of Canavan Disease. (2013) Stratford, NJ, UMDNJ/SOM Research Day. 75. Manzoor A., Francis J., Markov V., Shah Z., Leone P. Triheptanoin Treatment in the Nur7 Mouse Model of Canavan Disease. (2013) Glassboro, NJ. STEM-ROWAN Univ. 76. Assadi-IChansari M., Velazquez-Rodriguez Y., Zerafati G., and Leone P. The Phenotypic Differences between Probable Migraine and Definite Migraine. (2013) P03-017, San Diego, AAN 77.Assadi-IChansari M., Velazquez-Rodriguez Y., Zerafati G., and Leone P. Elevated Cerebral Lactate Levels on Multi-Voxel Proton MR Spectroscopy in Metachromatic Leukodystrophy. (2013) P03.016, San Diego, AAN 21of 22 EFTA01110727 LEONE, Pooke 78. Assadi-IChansari M., Velazquez-Rodriguez Y., Zerafati G., and Leone P. Do Genetic Factors Determine the Phenotypic Severity of Migraine? (2013). P06.155, San Diego, AAN Patents: Leone, P., Sorgi, F., and During, M.J. (1997) Delivery of Genes in the Central Nervous System. Tavazzi. B., Leone, P., Lazzarino (2005) Analytical Kits for the Chemical Diagnosis of Leukodystrophies & Non-hereditary Brain Patologies, Organic Acidosis, Metabolic Abnormalities of Purine & Pyrimidines, Congenital Metabolic Diseases Vitamin C Deficiency & Abnormal Oxidative States. Leone, P., Francis, JS (2012) PCT & US Metabolic Therapy of Neurodegenerative Diseases via the Catabolism of NAA. Extracurricular Activities 1) Photographs displayed at the American Museum of Natural History, New York, NY. Genomic Revolution Exhibit, May 26. 2001 -January Td, 2002; Museum of Natural History, North Carolina. Genomic Revolution Exhibit, April 26th, January Td, 2003; Fembank Museum of Natural History, Atlanta, GA, June 12th 2004 - January r d, 2005. 2) Editor of the documentary entitled " Little Heroes" , 2013 22of 22 EFTA01110728