Preclinical Potency and Safety Studies of an AAV2-Mediated Gene Therapy Vector for the Treatment of MERTK Associated Retinitis Pigmentosa

Proof of concept for MERTK gene replacement therapy has been demonstrated using different viral vectors in the Royal College of Surgeon (RCS) rat, a well characterized model of recessive retinitis pigmentosa that contains a mutation in the Mertk gene. MERTK plays a key role in renewal of photorecept...

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Hlavní autoři: Conlon, Thomas J., Deng, Wen-Tao, Erger, Kirsten, Cossette, Travis, Pang, Ji-jing, Ryals, Renee, Clément, Nathalie, Cleaver, Brian, McDoom, Issam, Boye, Shannon E., Peden, Marc C., Sherwood, Mark B., Abernathy, Corinne R., Alkuraya, Fowzan S., Boye, Sanford L., Hauswirth, William W.
Médium: Artigo
Jazyk:Inglês
Vydáno: Mary Ann Liebert, Inc. 2013
Témata:
Research Articles
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC3856558/
https://ncbi.nlm.nih.gov/pubmed/23692380
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/humc.2013.037
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