AKT3 Gene Transfer Promotes Anabolic Reprogramming and Photoreceptor Neuroprotection in a Pre-clinical Model of Retinitis Pigmentosa

Mutations within over 250 known genes are associated with inherited retinal degeneration. Clinical success following gene-replacement therapy for congenital blindness due to RPE65 mutations establishes a platform for the development of downstream treatments targeting other forms of inherited ocular...

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Udgivet i:Mol Ther
Main Authors: McDougald, Devin S., Papp, Tyler E., Zezulin, Alexandra U., Zhou, Shangzhen, Bennett, Jean
Format: Artigo
Sprog:Inglês
Udgivet: American Society of Gene & Cell Therapy 2019
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Original Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6612630/
https://ncbi.nlm.nih.gov/pubmed/31043342
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2019.04.009
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