Preclinical Dose-Escalation Study of Intravitreal AAV-RS1 Gene Therapy in a Mouse Model of X-linked Retinoschisis: Dose-Dependent Expression and Improved Retinal Structure and Function

Gene therapy for inherited retinal diseases has been shown to ameliorate functional and structural defects in both animal models and in human clinical trials. X-linked retinoschisis (XLRS) is an early-age onset macular dystrophy resulting from loss of an extracellular matrix protein (RS1). In prepar...

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Publicado en:Hum Gene Ther
Autores principales: Bush, Ronald A., Zeng, Yong, Colosi, Peter, Kjellstrom, Sten, Hiriyanna, Suja, Vijayasarathy, Camasamudram, Santos, Maria, Li, Jinbo, Wu, Zhijian, Sieving, Paul A.
Formato: Artigo
Lenguaje:Inglês
Publicado: Mary Ann Liebert, Inc. 2016
Materias:
Research Articles
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC4840830/
https://ncbi.nlm.nih.gov/pubmed/27036983
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2015.142
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