Safety and Efficacy of AAV5 Vectors Expressing Human or Canine CNGB3 in CNGB3-Mutant Dogs

Achromatopsia is an inherited retinal disorder of cone photoreceptors characterized by markedly reduced visual acuity, extreme light sensitivity, and absence of color discrimination. Approximately 50% of cases are caused by mutations in the cone photoreceptor-specific cyclic nucleotide gated channel...

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Veröffentlicht in:Hum Gene Ther Clin Dev
Hauptverfasser: Ye, Guo-jie, Komáromy, András M., Zeiss, Caroline, Calcedo, Roberto, Harman, Christine D., Koehl, Kristin L., Stewart, Gabriel A., Iwabe, Simone, Chiodo, Vince A., Hauswirth, William W., Aguirre, Gustavo D., Chulay, Jeffrey D.
Format: Artigo
Sprache:Inglês
Veröffentlicht: Mary Ann Liebert, Inc. 2017
Schlagworte:
Research Articles
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5733651/
https://ncbi.nlm.nih.gov/pubmed/29020838
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/humc.2017.125
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