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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. Sci Transl Med. 2012 Dec 19;4(165):165ra163. doi: 10.1126/scitranslmed.3003454. Sci Transl Med. 2012. PMID: 23253610 Free PMC article.
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. Among authors: bilaniuk l. Hum Gene Ther. 2002 Jul 20;13(11):1391-412. doi: 10.1089/104303402760128612. Hum Gene Ther. 2002. PMID: 12162821
Lithium citrate for Canavan disease.
Janson CG, Assadi M, Francis J, Bilaniuk L, Shera D, Leone P. Janson CG, et al. Among authors: bilaniuk l. Pediatr Neurol. 2005 Oct;33(4):235-43. doi: 10.1016/j.pediatrneurol.2005.04.015. Pediatr Neurol. 2005. PMID: 16194720
Serial 1H-MRS in GM2 gangliosidoses.
Assadi M, Baseman S, Janson C, Wang DJ, Bilaniuk L, Leone P. Assadi M, et al. Among authors: bilaniuk l. Eur J Pediatr. 2008 Mar;167(3):347-52. doi: 10.1007/s00431-007-0469-0. Epub 2007 Mar 27. Eur J Pediatr. 2008. PMID: 17387512
273 results