Hematopoietic Tumors in a Mouse Model of X-linked Chronic Granulomatous Disease after Lentiviral Vector-Mediated Gene Therapy.

Animals Disease Models, Animal Genetic Therapy / adverse effects Genetic Vectors / administration & dosage Granulomatous Disease, Chronic / complications Hematologic Neoplasms / etiology Humans Lentivirus / genetics Mice NADPH Oxidase 2 / genetics Time Factors Treatment Outcome

GLP Good Laboratory Practice X-linked chronic granulomatosis disease gene therapy inflammation lentiviral vectors mouse model myelodysplastic syndrome

Journal

Molecular therapy : the journal of the American Society of Gene Therapy

ISSN: 1525-0024

Titre abrégé: Mol Ther

Pays: United States

ID NLM: 100890581

Informations de publication

Date de publication:
06 01 2021

Historique:

received: 10 04 2020

revised: 03 08 2020

accepted: 20 09 2020

pubmed: 4 10 2020

medline: 1 10 2021

entrez: 3 10 2020

Statut: ppublish

Résumé

Chronic granulomatous disease (CGD) is a rare inherited disorder due to loss-of-function mutations in genes encoding the NADPH oxidase subunits. Hematopoietic stem and progenitor cell (HSPC) gene therapy (GT) using regulated lentiviral vectors (LVs) has emerged as a promising therapeutic option for CGD patients. We performed non-clinical Good Laboratory Practice (GLP) and laboratory-grade studies to assess the safety and genotoxicity of LV targeting myeloid-specific Gp91

Identifiants

DOI: 10.1016/j.ymthe.2020.09.030 PMID: 33010230 PMC: PMC7791081

pubmed: 33010230

pii: S1525-0016(20)30487-1

doi: 10.1016/j.ymthe.2020.09.030

pmc: PMC7791081

pii:

doi:

Substances chimiques

NADPH Oxidase 2 EC 1.6.3.-

Types de publication

Journal Article Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

Pagination

86-102

Informations de copyright

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