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Immune responses to AAV in a phase I study for Canavan disease.
McPhee SW, Janson CG, Li C, Samulski RJ, Camp AS, Francis J, Shera D, Lioutermann L, Feely M, Freese A, Leone P. McPhee SW, et al. J Gene Med. 2006 May;8(5):577-88. doi: 10.1002/jgm.885. J Gene Med. 2006. PMID: 16532510 Clinical Trial.
Clinical protocol. Gene therapy of Canavan disease: AAV-2 vector for neurosurgical delivery of aspartoacylase gene (ASPA) to the human brain.
Janson C, McPhee S, Bilaniuk L, Haselgrove J, Testaiuti M, Freese A, Wang DJ, Shera D, Hurh P, Rupin J, Saslow E, Goldfarb O, Goldberg M, Larijani G, Sharrar W, Liouterman L, Camp A, Kolodny E, Samulski J, Leone P. Janson C, et al. Hum Gene Ther. 2002 Jul 20;13(11):1391-412. doi: 10.1089/104303402760128612. Hum Gene Ther. 2002. PMID: 12162821
Long-term follow-up after gene therapy for canavan disease.
Leone P, Shera D, McPhee SW, Francis JS, Kolodny EH, Bilaniuk LT, Wang DJ, Assadi M, Goldfarb O, Goldman HW, Freese A, Young D, During MJ, Samulski RJ, Janson CG. Leone P, et al. Among authors: mcphee sw. Sci Transl Med. 2012 Dec 19;4(165):165ra163. doi: 10.1126/scitranslmed.3003454. Sci Transl Med. 2012. PMID: 23253610 Free PMC article.
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