Lessons Learned from the Development of the First FDA-Approved Gene Therapy Drug, Voretigene Neparvovec-rzyl

Author:

Bennett Jean,Maguire Albert M.

Publisher

Cold Spring Harbor Laboratory

Subject

General Biochemistry, Genetics and Molecular Biology

Reference61 articles.

1. 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

2. Adverum Biotechnologies 2020. Adverum biotechnologies reports additional clinical data from first cohort of OPTIC Phase 1 Trial of ADVM-022 intravitreal gene therapy for Wet AMD at the Atlantic Coast Retina Club Macula 20/20 Annual Meeting. GlobeNewswire, January 11, 2020. https://www.globenewswire.com/news-release/2020/01/11/1969182/0/en/Adverum-Biotechnologies-Reports-Additional-Clinical-Data-from-First-Cohort-of-OPTIC-Phase-1-Trial-of-ADVM-022-Intravitreal-Gene-Therapy-for-Wet-AMD-at-the-Atlantic-Coast-Retina-Clu.html

3. Congenital stationary night blindness in the dog: common mutation in the RPE65 gene indicates founder effect;Mol Vis,1998

4. A Virtual Reality Orientation and Mobility Test for Inherited Retinal Degenerations: Testing a Proof-of-Concept After Gene Therapy

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