Safety and Biodistribution Evaluation in Cynomolgus Macaques of rAAV2tYF-PR1.7-hCNGB3, a Recombinant AAV Vector for Treatment of Achromatopsia

Applied Genetic Technologies Corporation (AGTC) is developing rAAV2tYF-PR1.7-hCNGB3, a recombinant adeno-associated viral (rAAV) vector expressing the human CNGB3 gene, for treatment of achromatopsia, an inherited retinal disorder characterized by markedly reduced visual acuity, extreme light sensit...

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Détails bibliographiques
Publié dans:Hum Gene Ther Clin Dev
Auteurs principaux: Ye, Guo-jie, Budzynski, Ewa, Sonnentag, Peter, Nork, T. Michael, Miller, Paul E., Sharma, Alok K., Ver Hoeve, James N., Smith, Leia M., Arndt, Tara, Calcedo, Roberto, Gaskin, Chantelle, Robinson, Paulette M., Knop, David R., Hauswirth, William W., Chulay, Jeffrey D.
Format: Artigo
Langue:Inglês
Publié: Mary Ann Liebert, Inc. 2016
Sujets:
Research Articles
Accès en ligne:https://ncbi.nlm.nih.gov/pmc/articles/PMC4851175/
https://ncbi.nlm.nih.gov/pubmed/27003753
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/humc.2015.164
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