|
Bennett, Jean (2017) Taking Stock of Retinal Gene Therapy: Looking Back and Moving Forward. Mol Ther 25:1076-1094
|
|
Jacobs, Jonathan B; Dell'Osso, Louis F; Wang, Zhong I et al. (2009) Using the NAFX to measure the effectiveness over time of gene therapy in canine LCA. Invest Ophthalmol Vis Sci 50:4685-92
|
|
Maguire, Albert M; High, Katherine A; Auricchio, Alberto et al. (2009) Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial. Lancet 374:1597-605
|
|
Lebherz, Corinna; Maguire, Albert; Tang, Waixing et al. (2008) Novel AAV serotypes for improved ocular gene transfer. J Gene Med 10:375-82
|
|
Bennicelli, Jeannette; Wright, John Fraser; Komaromy, Andras et al. (2008) Reversal of blindness in animal models of leber congenital amaurosis using optimized AAV2-mediated gene transfer. Mol Ther 16:458-65
|
|
Maguire, Albert M; Simonelli, Francesca; Pierce, Eric A et al. (2008) Safety and efficacy of gene transfer for Leber's congenital amaurosis. N Engl J Med 358:2240-8
|
|
Allocca, Mariacarmela; Doria, Monica; Petrillo, Marco et al. (2008) Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice. J Clin Invest 118:1955-64
|
|
Endo, Masayuki; Zoltick, Philip W; Chung, Daniel C et al. (2007) Gene transfer to ocular stem cells by early gestational intraamniotic injection of lentiviral vector. Mol Ther 15:579-87
|
|
Jacobs, Jonathan B; Dell'Osso, Louis F; Hertle, Richard W et al. (2006) Eye movement recordings as an effectiveness indicator of gene therapy in RPE65-deficient canines: implications for the ocular motor system. Invest Ophthalmol Vis Sci 47:2865-75
|
|
Bedrosian, Jeffrey C; Gratton, Michael Anne; Brigande, John V et al. (2006) In vivo delivery of recombinant viruses to the fetal murine cochlea: transduction characteristics and long-term effects on auditory function. Mol Ther 14:328-35
|
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