论文信息 - Seeing the Light

Seeing the Light

One-time gene therapy resulted in multiyear visual improvement, and new approaches were used to evaluate effects on retinal structure. One-time gene therapy resulted in multiyear visual improvement, and new approaches were used to evaluate effects on retinal structure.

Jean Bennett | Eric A. Pierce | J. Bennett | Adam P. Wojno | E. Pierce

[1] Hemant Khanna,et al. Gene therapy rescues photoreceptor blindness in dogs and paves the way for treating human X-linked retinitis pigmentosa , 2012, Proceedings of the National Academy of Sciences.

[2] David Williams,et al. Noninvasive imaging of the human rod photoreceptor mosaic using a confocal adaptive optics scanning ophthalmoscope , 2011, Biomedical optics express.

[3] Alexander Sumaroka,et al. Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement , 2013, Proceedings of the National Academy of Sciences.

[4] Jean Bennett,et al. Gene therapy restores vision in a canine model of childhood blindness , 2001, Nature Genetics.

[5] W. Hauswirth,et al. Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindness. , 2005, Molecular therapy : the journal of the American Society of Gene Therapy.

[6] William J Feuer,et al. Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years. , 2012, Archives of ophthalmology.

[7] G. G. Stokes. "J." , 1890, The New Yale Book of Quotations.

[8] W. Hauswirth,et al. Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial. , 2008, Human gene therapy.