Technique of retinal gene therapy: delivery of viral vector into the subretinal space
Author:
Publisher
Springer Science and Business Media LLC
Subject
Ophthalmology
Link
http://www.nature.com/articles/eye2017158.pdf
Reference18 articles.
1. Jacobson SG, Cideciyan AV, Roman AJ, Sumaroka A, Schwartz SB, Heon E et al. Improvement and decline in vision with gene therapy in childhood blindness. N Engl J Med 2015; 372: 1920–1926.
2. Bainbridge JW, Mehat MS, Sundaram V, Robbie SJ, Barker SE, Ripamonti C et al. Long-term effect of gene therapy on Leber's congenital amaurosis. N Engl J Med 2015; 372: 1887–1897.
3. Edwards TL, Jolly JK, Groppe M, Barnard AR, Cottriall CL, Tolmachova T et al. Visual Acuity after Retinal Gene Therapy for Choroideremia. N Engl J Med 2016; 374: 1996–1998.
4. Bennett J, Wellman J, Marshall KA, McCague S, Ashtari M, DiStefano-Pappas J et al. Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial. Lancet 2016; 388: 661–672.
5. Ghazi NG, Abboud EB, Nowilaty SR, Alkuraya H, Alhommadi A, Cai H et al. Treatment of retinitis pigmentosa due to MERTK mutations by ocular subretinal injection of adeno-associated virus gene vector: results of a phase I trial. Hum Genet 2016; 135: 327–343.
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