Immune function in X-linked retinoschisis subjects in an AAV8-RS1 phase I/IIa gene therapy trial

This study explored systemic immune changes in 11 subjects with X-linked retinoschisis (XLRS) in a phase I/IIa adeno-associated virus 8 (AAV8)-RS1 gene therapy trial (ClinicalTrials.gov: NCT02317887). Immune cell proportions and serum analytes were compared to 12 healthy male controls. At pre-dosing...

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Опубликовано в: :Mol Ther
Главные авторы: Mishra, Alaknanda, Vijayasarathy, Camasamudram, Cukras, Catherine A., Wiley, Henry E., Sen, H. Nida, Zeng, Yong, Wei, Lisa L., Sieving, Paul A.
Формат: Artigo
Язык:Inglês
Опубликовано: American Society of Gene & Cell Therapy 2021
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Original Article
Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC8178519/
https://ncbi.nlm.nih.gov/pubmed/33601057
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2021.02.013
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