Retinal AAV8-RS1 Gene Therapy for X-Linked Retinoschisis: Initial Findings from a Phase I/IIa Trial by Intravitreal Delivery

This study evaluated the safety and tolerability of ocular RS1 adeno-associated virus (AAV8-RS1) gene augmentation therapy to the retina of participants with X-linked retinoschisis (XLRS). XLRS is a monogenic trait affecting only males, caused by mutations in the RS1 gene. Retinoschisin protein is s...

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Detalhes bibliográficos
Publicado no:Mol Ther
Main Authors: Cukras, Catherine, Wiley, Henry E., Jeffrey, Brett G., Sen, H. Nida, Turriff, Amy, Zeng, Yong, Vijayasarathy, Camasamudram, Marangoni, Dario, Ziccardi, Lucia, Kjellstrom, Sten, Park, Tae Kwon, Hiriyanna, Suja, Wright, J. Fraser, Colosi, Peter, Wu, Zhijian, Bush, Ronald A., Wei, Lisa L., Sieving, Paul A.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2018
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6127971/
https://ncbi.nlm.nih.gov/pubmed/30196853
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2018.05.025
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