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

doi:10.1016/j.ymthe.2021.02.013

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Article

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

Molecular therapy : the journal of the American Society of Gene Therapy - 2 Jun 2021

Mishra Alaknanda, Vijayasarathy Camasamudram, Cukras Catherine A, Wiley Henry E, Sen H Nida, Zeng Yong, Wei Lisa L, Sieving Paul A

Abstract excerpt

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 baseline the mean CD4/CD8 ratio of XLRS subjects was elevated. CD11c+ myeloid dendritic cells (DCs) and the serum...

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Identifiers

PMID: 33601057
DOI: 10.1016/j.ymthe.2021.02.013
PMCID: PMC8178519

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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