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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Dettagli Bibliografici
Pubblicato in:Mol Ther
Autori principali: 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.
Natura: Artigo
Lingua:Inglês
Pubblicazione: American Society of Gene & Cell Therapy 2018
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Original Article
Accesso online: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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