Safety and Biodistribution Evaluation in Cynomolgus Macaques of rAAV2tYF-CB-hRS1, a Recombinant Adeno-Associated Virus Vector Expressing Retinoschisin

Applied Genetic Technologies Corporation is developing rAAV2tYF-CB-hRS1, a recombinant adeno-associated virus (rAAV) vector for treatment of X-linked retinoschisis (XLRS), an inherited retinal disease characterized by splitting (schisis) of retinal layers causing poor vision. We report here results...

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Библиографические подробности
Опубликовано в: :Hum Gene Ther Clin Dev
Главные авторы: Ye, Guo-Jie, Budzynski, Ewa, Sonnentag, Peter, Miller, Paul E., Sharma, Alok K., Ver Hoeve, James N., Howard, Kellie, Knop, David R., Neuringer, Martha, McGill, Trevor, Stoddard, Jonathan, Chulay, Jeffrey D.
Формат: Artigo
Язык:Inglês
Опубликовано: Mary Ann Liebert, Inc. 2015
Предметы:
Clinical Protocols
Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC4788136/
https://ncbi.nlm.nih.gov/pubmed/26390090
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/humc.2015.076
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