Busulfan Combined with Immunosuppression Allows Efficient Engraftment of Gene-Modified Cells in a Rhesus Macaque Model.
Animals
Busulfan
/ pharmacology
Gene Expression
Genes, Reporter
Genetic Therapy
/ methods
Genetic Vectors
/ genetics
Hematopoietic Stem Cell Transplantation
/ methods
Hematopoietic Stem Cells
/ metabolism
Immunosuppressive Agents
/ pharmacology
Macaca mulatta
Models, Animal
Transduction, Genetic
Transgenes
Transplantation Conditioning
gamma-Globins
/ genetics
busulfan conditioning
gene therapy
hematopoietic stem cells
immunological rejection
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:
04 09 2019
04 09 2019
Historique:
received:
19
12
2018
revised:
13
05
2019
accepted:
29
05
2019
pubmed:
30
6
2019
medline:
17
6
2020
entrez:
30
6
2019
Statut:
ppublish
Résumé
Busulfan conditioning is utilized for hematopoietic stem cell (HSC) depletion in the context of HSC gene-therapy conditioning but may result in insufficient immunosuppression. In this study, we evaluated whether additional immunosuppression is required for efficient engraftment of gene-modified cells using a rhesus HSC lentiviral gene-therapy model. We transduced half of rhesus CD34
Identifiants
pubmed: 31253582
pii: S1525-0016(19)30269-2
doi: 10.1016/j.ymthe.2019.05.022
pmc: PMC6731177
pii:
doi:
Substances chimiques
Immunosuppressive Agents
0
gamma-Globins
0
Busulfan
G1LN9045DK
Types de publication
Journal Article
Research Support, N.I.H., Intramural
Langues
eng
Sous-ensembles de citation
IM
Pagination
1586-1596Subventions
Organisme : NHLBI NIH HHS
ID : R01 HL095791
Pays : United States
Organisme : NIAID NIH HHS
ID : U19 AI051731
Pays : United States
Organisme : NHLBI NIH HHS
ID : U19 HL129902
Pays : United States
Informations de copyright
Published by Elsevier Inc.
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