Immune responses to AAV in a phase I study for Canavan disease
McPhee, S. W. J., Janson, C. G., Li, C., Samulski, R. J., Camp, A. S., Francis, J., Shera, D., Lioutermann, L., Feely, M., Freese, A., Leone, P.
Published in The journal of gene medicine (01.05.2006)
Published in The journal of gene medicine (01.05.2006)
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Journal Article
Quantitative comparison of expression with adeno-associated virus (AAV-2) brain-specific gene cassettes
XU, R, JANSON, C. G, LEONE, P, CHEN, Q, DICKER, B, DURING, M. J, MASTAKOV, M, LAWLOR, P, YOUNG, D, MOURAVLEV, A, FITZSIMONS, H, CHOI, K-L, MA, H, DRAGUNOW, M
Published in Gene therapy (01.09.2001)
Published in Gene therapy (01.09.2001)
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Journal Article
Aspartoacylase gene transfer to the mammalian central nervous system with therapeutic implications for Canavan disease
Leone, P., Janson, C. G., Bilianuk, L., Wang, Z., Sorgi, F., Huang, L., Matalon, R., Kaul, R., Zeng, Z., Freese, A., McPhee, S. W., Mee, E., During, M. J.
Published in Annals of neurology (01.07.2000)
Published in Annals of neurology (01.07.2000)
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Journal Article
Natural history of Canavan disease revealed by proton magnetic resonance spectroscopy (1H-MRS) and diffusion-weighted MRI
Janson, C G, McPhee, S W J, Francis, J, Shera, D, Assadi, M, Freese, A, Hurh, P, Haselgrove, J, Wang, D J, Bilaniuk, L, Leone, P
Published in Neuropediatrics (01.08.2006)
Published in Neuropediatrics (01.08.2006)
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Journal Article
Effects of AAV-2-mediated aspartoacylase gene transfer in the tremor rat model of Canavan disease
McPhee, S.W.J., Francis, J., Janson, C.G., Serikawa, T., Hyland, K., Ong, E.O., Raghavan, S.S., Freese, A., Leone, P.
Published in Brain research. Molecular brain research. (27.04.2005)
Published in Brain research. Molecular brain research. (27.04.2005)
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Journal Article
Functional regulatory regions of human transcription factor MEF2C
Janson, C.G, Chen, Y, Li, Y, Leifer, D
Published in Brain research. Molecular brain research. (16.12.2001)
Published in Brain research. Molecular brain research. (16.12.2001)
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Journal Article
Multi-site partitioned delivery of human tyrosine hydroxylase gene with phenotypic recovery in Parkinsonian rats
Leone, P, McPhee, S W, Janson, C G, Davidson, B L, Freese, A, During, M J
Published in Neuroreport (27.04.2000)
Published in Neuroreport (27.04.2000)
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Journal Article
Viral-based gene transfer to the mammalian CNS for functional genomic studies
Janson, Christopher G., McPhee, Scott W.J., Leone, Paola, Freese, Andrew, During, Matthew J.
Published in Trends in Neurosciences (01.12.2001)
Published in Trends in Neurosciences (01.12.2001)
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Book Review
Journal Article
Long-term follow-up after gene therapy for canavan disease
Leone, Paola, Shera, David, McPhee, Scott W J, Francis, Jeremy S, Kolodny, Edwin H, Bilaniuk, Larissa T, Wang, Dah-Jyuu, Assadi, Mitra, Goldfarb, Olga, Goldman, H Warren, Freese, Andrew, Young, Deborah, During, Matthew J, Samulski, R Jude, Janson, Christopher G
Published in Science translational medicine (19.12.2012)
Published in Science translational medicine (19.12.2012)
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Journal Article
Viral Vectors as Part of an Integrated Functional Genomics Program
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Book Review
Journal Article