Directed Evolution of AAV Targeting Primate Retina by Intravitreal Injection Identifies R100, a Variant Demonstrating Robust Gene Delivery and Therapeutic Efficacy in Non-Human Primates

Autor: Kotterman M, D Kirn, Holt J, Christopher A. Schmitt, P Szymanski, M Quezada, T Vazin, Roxanne H. Croze, Hassanipour M, G. Beliakoff, K. Barglow, David V. Schaffer, Francis P, Leong M

Targeted AAV vectors are needed for safe and efficient delivery to and transduction of specific tissue target(s) in patients. Effective intravitreal delivery for retina gene therapy is not feasible with wildtype AAV. We employed directed evolution in

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::16d856b18df0a4914bced918e54fd9d6
https://doi.org/10.1101/2021.06.24.449775

P1516Novel cardiotropic AAV variant C102 vectors show superior gene delivery & reduced immunogenicity in non-human primates, transduction of human cardiomyocytes, & correction of Fabry disease phenotype

Autor: Roxanne H. Croze, J. Nye, P Szymanski, Francis P, K. Whittlesey, Hassanipour M, L Johnson, G. Beliakoff, T Vazin, D Schaffer, Kotterman M, Christopher A. Schmitt, G Brooks, M Quezada, D Kirn

Publikováno v: European Heart Journal. 40

Background Cardiac-targeted gene therapy vectors are needed. Fabry disease is a rare, X-linked disorder caused by mutations in the GLA gene, which encodes α-galactosidase A (GLA). Storage and accumulation of glycolipid substrates of GLA leads to org

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::f73bb121e32ebb88580811a8be2184d8
https://doi.org/10.1093/eurheartj/ehz748.0278

Design and Characterization of a Novel Intravitreal Dual-Transgene Genetic Medicine for Neovascular Retinopathies.

Autor: Calton MA; 4D Molecular Therapeutics, Emeryville, California, United States., Croze RH; 4D Molecular Therapeutics, Emeryville, California, United States., Burns C; 4D Molecular Therapeutics, Emeryville, California, United States., Beliakoff G; 4D Molecular Therapeutics, Emeryville, California, United States., Vazin T; 4D Molecular Therapeutics, Emeryville, California, United States., Szymanski P; 4D Molecular Therapeutics, Emeryville, California, United States., Schmitt C; 4D Molecular Therapeutics, Emeryville, California, United States., Klein A; 4D Molecular Therapeutics, Emeryville, California, United States., Leong M; 4D Molecular Therapeutics, Emeryville, California, United States., Quezada M; 4D Molecular Therapeutics, Emeryville, California, United States., Holt J; 4D Molecular Therapeutics, Emeryville, California, United States., Bolender G; 4D Molecular Therapeutics, Emeryville, California, United States., Barglow K; 4D Molecular Therapeutics, Emeryville, California, United States., Khoday D; 4D Molecular Therapeutics, Emeryville, California, United States., Mason T; 4D Molecular Therapeutics, Emeryville, California, United States., Delaria K; 4D Molecular Therapeutics, Emeryville, California, United States., Hassanipour M; 4D Molecular Therapeutics, Emeryville, California, United States., Kotterman M; 4D Molecular Therapeutics, Emeryville, California, United States., Khanani AM; Sierra Eye Associates, Reno, Nevada, United States.; University of Nevada, Reno School of Medicine, Reno, Nevada, United States., Schaffer D; University of California, Berkeley, California, United States., Francis P; 4D Molecular Therapeutics, Emeryville, California, United States., Kirn D; 4D Molecular Therapeutics, Emeryville, California, United States.; University of California, Berkeley, California, United States.

Publikováno v: Investigative ophthalmology & visual science [Invest Ophthalmol Vis Sci] 2024 Dec 02; Vol. 65 (14), pp. 1.