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Safety and efficacy evaluations of an adeno-associated virus variant for preparing IL10-secreting human neural stem cell-based therapeutics

Authors
 Mira Cho  ;  Kwangsoo Jung  ;  Seung-Hyun Kim  ;  Il-Sun Kim  ;  Miri Kim  ;  Mikyung Shin  ;  Haeshin Lee  ;  Kook In Park  ;  Jae-Hyung Jang 
Citation
 GENE THERAPY, Vol.26(5) : 135-150, 2019 
Journal Title
 GENE THERAPY 
ISSN
 0969-7128 
Issue Date
2019
Abstract
Gene therapy technologies are inevitably required to boost the therapeutic performance of cell therapies; thus, validating the efficacy of gene carriers specifically used for preparing cellular therapeutics is a prerequisite for evaluating the therapeutic capabilities of gene and cell combinatorial therapies. Herein, the efficacy of a recombinant adeno-associated virus derivative (rAAVr3.45) was examined to evaluate its potential as a gene carrier for genetically manipulating interleukin-10 (IL10)-secreting human neural stem cells (hNSCs) that can potentially treat ischemic injuries or neurological disorders. Safety issues that could arise during the virus preparation or viral infection were investigated; no replication-competent AAVs were detected in the final cell suspensions, transgene expression was mostly transient, and no severe interference on endogenous gene expression by viral infection occurred. IL10 secretion from hNSCs infected by rAAVr3.45 encoding IL10 did not alter the transcriptional profile of any gene by more than threefold, but the exogenously boosted IL10 was sufficient to provoke immunomodulatory effects in an ischemic brain injury animal model, thereby accelerating the recovery of neurological deficits and the reduction of brain infarction volume. This study presents evidence that rAAVr3.45 can be potentially used as a gene carrier to prepare stem cell therapeutics.
Full Text
https://www.nature.com/articles/s41434-019-0057-8
DOI
10.1038/s41434-019-0057-8
Appears in Collections:
1. College of Medicine (의과대학) > Dept. of Pediatrics (소아청소년과학교실) > 1. Journal Papers
Yonsei Authors
Kim, Mi Ri(김미리) ORCID logo https://orcid.org/0000-0002-0380-1677
Park, Kook In(박국인) ORCID logo https://orcid.org/0000-0001-8499-9293
URI
https://ir.ymlib.yonsei.ac.kr/handle/22282913/170074
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