AAV-mediated RLBP1 gene therapy improves the rate of dark adaptation in Rlbp1 knockout mice

Recessive mutations in RLBP1 cause a form of retinitis pigmentosa in which the retina, before its degeneration leads to blindness, abnormally slowly recovers sensitivity after exposure to light. To develop a potential gene therapy for this condition, we tested multiple recombinant adeno-associated v...

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Main Authors: Vivian W Choi, Chad E Bigelow, Terri L McGee, Akshata N Gujar, Hui Li, Shawn M Hanks, Joanna Vrouvlianis, Michael Maker, Barrett Leehy, Yiqin Zhang, Jorge Aranda, George Bounoutas, John T Demirs, Junzheng Yang, Richard Ornberg, Yu Wang, Wendy Martin, Kelly R Stout, Gregory Argentieri, Paul Grosenstein, Danielle Diaz, Oliver Turner, Bruce D Jaffee, Seshidhar R Police, Thaddeus P Dryja
Format: Artigo
Jezik:Inglês
Izdano: Elsevier 2015-01-01
Serija:Molecular Therapy: Methods & Clinical Development
Online dostop:http://www.sciencedirect.com/science/article/pii/S2329050116300341
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