Safety and Biodistribution Evaluation in CNGB3-Deficient Mice 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 virus (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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Gepubliceerd in:Hum Gene Ther Clin Dev
Hoofdauteurs: Ye, Guo-jie, Budzynski, Ewa, Sonnentag, Peter, Nork, T. Michael, Miller, Paul E., McPherson, Leslie, Ver Hoeve, James N., Smith, Leia M., Arndt, Tara, Mandapati, Savitri, Robinson, Paulette M., Calcedo, Roberto, Knop, David R., Hauswirth, William W., Chulay, Jeffrey D.
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: Mary Ann Liebert, Inc. 2016
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Research Articles
Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4851180/
https://ncbi.nlm.nih.gov/pubmed/27003752
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/humc.2015.163
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