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
By W. Hauswirth, T. Aleman, S. Kaushal, A. Cideciyan, S. Schwartz, Lili Wang, T. Conlon, S. Boye, T. Flotte, B. Byrne and 1 more. Human Gene Therapy, 2008
999 citations recorded by the source corpus.
Subfields: Molecular Biology, Ophthalmology
View Paper: https://www.semanticscholar.org/paper/5c2432711238fe7b0ce0a4b8ae361f6df826c8f9
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In Ophthalmology
Citations in the corpus
- Gene therapy clinical trials worldwide to 2012 – an update (2013, background): “Using sub-retinal administration of recombinant adeno-associated viral vectors (rAAV) expressing RPE65, three independent clinical trials for LCA have been initiated [32–35].”
- Gene Therapy for Leber Congenital Amaurosis caused by RPE65 mutations: Safety and Efficacy in Fifteen Children and Adults Followed up to Three Years (2011, methodology): “Best-corrected visual acuity (VA) was measured using Early Treatment Diabetic Retinopathy Study methodology(28) at all baseline and postoperative visits.(17,20) Best-corrected VA was scored as the number of letters correctly read after adjusting for distance and expressed as logMAR.”
- Non‐syndromic retinitis pigmentosa (2018, positive): “of using RPE65 gene augmentation therapy in patients with LCA or early-onset RP due to bi-allelic RPE65 mutations were reported (Bainbridge et al., 2008; Hauswirth et al., 2008; Maguire et al., 2008).”