A Novel BaEVRless-Pseudotyped γ-Globin Lentiviral Vector Drives High and Stable Fetal Hemoglobin Expression and Improves Thalassemic Erythropoiesis
Erythropoiesis
/ genetics
Fetal Hemoglobin
/ genetics
Gene Expression
Gene Order
Gene Transfer Techniques
Genetic Engineering
Genetic Therapy
/ methods
Genetic Vectors
/ genetics
Hematopoietic Stem Cells
/ metabolism
Humans
Lentivirus
/ genetics
Recombination, Genetic
Transduction, Genetic
Transgenes
beta-Thalassemia
/ genetics
gamma-Globins
/ genetics
BaEVRless
HbF
envelope glycoproteins
gene therapy
β-thalassemia
γ-globin lentiviral vector
Journal
Human gene therapy
ISSN: 1557-7422
Titre abrégé: Hum Gene Ther
Pays: United States
ID NLM: 9008950
Informations de publication
Date de publication:
05 2019
05 2019
Historique:
pubmed:
17
10
2018
medline:
13
3
2020
entrez:
17
10
2018
Statut:
ppublish
Résumé
It has previously been demonstrated that the self-inactivating γ-globin lentiviral vector GGHI can significantly increase fetal hemoglobin (HbF) in erythroid cells from thalassemia patients and thus improve the disease phenotype
Identifiants
pubmed: 30324804
doi: 10.1089/hum.2018.022
doi:
Substances chimiques
gamma-Globins
0
Fetal Hemoglobin
9034-63-3
Types de publication
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM