Sustained fetal hemoglobin induction in vivo is achieved by

Animals Erythroid Cells Fetal Hemoglobin / genetics Macaca mulatta Mice Receptors, Erythropoietin / genetics Repressor Proteins

Journal

Science translational medicine
ISSN: 1946-6242
Titre abrégé: Sci Transl Med
Pays: United States
ID NLM: 101505086

Informations de publication

Date de publication:
28 04 2021
Historique:
received: 25 01 2020
revised: 13 10 2020
accepted: 02 04 2021
entrez: 29 4 2021
pubmed: 30 4 2021
medline: 13 7 2021
Statut: ppublish

Résumé

Hematopoietic stem cell gene therapy for hemoglobin disorders, including sickle cell disease, requires high-efficiency lentiviral gene transfer and robust therapeutic globin expression in erythroid cells. Erythropoietin is a key cytokine for erythroid proliferation and differentiation (erythropoiesis), and truncated human erythropoietin receptors (thEpoR) have been reported in familial polycythemia. We reasoned that coexpression of thEpoR could enhance the phenotypic effect of a therapeutic vector in erythroid cells in xenograft mouse and autologous nonhuman primate transplantation models. We generated thEpoR by deleting 40 amino acids from the carboxyl terminus, allowing for erythropoietin-dependent enhanced erythropoiesis of gene-modified cells. We then designed lentiviral vectors encoding both thEpoR and B cell lymphoma/leukemia 11A (

Identifiants

DOI: 10.1126/scitranslmed.abb0411 PMID: 33910976
pubmed: 33910976
pii: 13/591/eabb0411
doi: 10.1126/scitranslmed.abb0411
pii:
doi:

Substances chimiques

Receptors, Erythropoietin 0
Repressor Proteins 0
Fetal Hemoglobin 9034-63-3

Types de publication

Journal Article Research Support, N.I.H., Extramural Research Support, N.I.H., Intramural

Langues

eng

Sous-ensembles de citation

IM

Informations de copyright

Copyright © 2021 The Authors, some rights reserved; exclusive licensee American Association for the Advancement of Science. No claim to original U.S. Government Works.

Auteurs

Naoya Uchida (N)

Cellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD 20892, USA. uchidan@nhlbi.nih.gov.
Division of Molecular and Medical Genetics, Center for Gene and Cell Therapy, The Institute of Medical Science, The University of Tokyo, Minato-ku, Tokyo 108-8639, Japan.
ORCID: 0000-0001-6322-8991

Francesca Ferrara (F)

Department of Hematology, St. Jude Children's Research Hospital (SJCRH), Memphis, TN 38105, USA.
ORCID: 0000-0002-4973-1520

Claire M Drysdale (CM)

Cellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD 20892, USA.
ORCID: 0000-0002-7673-441X

Morgan Yapundich (M)

Cellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD 20892, USA.
ORCID: 0000-0003-1016-2153

Jackson Gamer (J)

Cellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD 20892, USA.
ORCID: 0000-0002-6547-3568

Tina Nassehi (T)

Cellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD 20892, USA.
ORCID: 0000-0002-8576-9737

Julia DiNicola (J)

Cellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD 20892, USA.
ORCID: 0000-0001-7076-8259

Yoshitaka Shibata (Y)

Cellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD 20892, USA.
ORCID: 0000-0003-0107-2877

Matthew Wielgosz (M)

Department of Hematology, St. Jude Children's Research Hospital (SJCRH), Memphis, TN 38105, USA.
ORCID: 0000-0002-3247-9488

Yoon-Sang Kim (YS)

Department of Hematology, St. Jude Children's Research Hospital (SJCRH), Memphis, TN 38105, USA.
ORCID: 0000-0002-2590-8096

Matthew Bauler (M)

Vector Development and Production Laboratory, SJCRH, Memphis, TN 38105, USA.
ORCID: 0000-0003-1861-9971

Robert E Throm (RE)

Vector Development and Production Laboratory, SJCRH, Memphis, TN 38105, USA.
ORCID: 0000-0002-9191-4742

Juan J Haro-Mora (JJ)

Cellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD 20892, USA.
ORCID: 0000-0002-6679-7812

Selami Demirci (S)

Cellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD 20892, USA.

Aylin C Bonifacino (AC)

Translational Stem Cell Biology Branch, NHLBI, NIH, Bethesda, MD 20892, USA.

Allen E Krouse (AE)

Translational Stem Cell Biology Branch, NHLBI, NIH, Bethesda, MD 20892, USA.

N Seth Linde (NS)

Translational Stem Cell Biology Branch, NHLBI, NIH, Bethesda, MD 20892, USA.
ORCID: 0000-0002-5336-9691

Robert E Donahue (RE)

Cellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD 20892, USA.

Byoung Ryu (B)

Department of Hematology, St. Jude Children's Research Hospital (SJCRH), Memphis, TN 38105, USA.
Umoja Biopharma, 1920 Terry Ave., Seattle, WA 98101, USA.

John F Tisdale (JF)

Cellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD 20892, USA.
ORCID: 0000-0002-8535-9439

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Classifications MeSH

questionsmedicales.fr › Eucaryotes › Animaux › Sustained fetal hemoglobin induction in vivo...
questionsmedicales.fr › Cellules › Cellules érythroïdes › Sustained fetal hemoglobin induction in vivo...
questionsmedicales.fr › Acides aminés, peptides et protéines › Protéines › Hémoprotéines › Globines › Hémoglobines › Hémoglobine foetale › Sustained fetal hemoglobin induction in vivo...
questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Primates › Haplorhini › Catarrhini › Cercopithecidae › Cercopithecinae › Macaca › Macaca mulatta › Sustained fetal hemoglobin induction in vivo...
questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Rodentia › Muridae › Murinae › Souris › Sustained fetal hemoglobin induction in vivo...
questionsmedicales.fr › Acides aminés, peptides et protéines › Protéines › Protéines membranaires › Récepteurs de surface cellulaire › Récepteurs peptidiques › Récepteur facteur croissance › Récepteurs aux facteurs de croissance hématopoïétique › Récepteur érythropoïétine › Sustained fetal hemoglobin induction in vivo...
questionsmedicales.fr › Acides aminés, peptides et protéines › Protéines › Facteurs de transcription › Protéines de répression › Sustained fetal hemoglobin induction in vivo...