Hematopoietic-Stem-Cell-Targeted Gene-Addition and Gene-Editing Strategies for β-hemoglobinopathies.

CRISPR-Cas Systems / genetics Gene Editing Hematopoietic Stem Cell Transplantation Hematopoietic Stem Cells Hemoglobinopathies / genetics Humans
CRISPR-Cas9 gene therapy genome editing hematopoietic stem cells lentiviral vector sickle cell disease

Journal

Cell stem cell
ISSN: 1875-9777
Titre abrégé: Cell Stem Cell
Pays: United States
ID NLM: 101311472

Informations de publication

Date de publication:
04 02 2021
Historique:
entrez: 5 2 2021
pubmed: 6 2 2021
medline: 20 5 2021
Statut: ppublish

Résumé

Sickle cell disease (SCD) is caused by a well-defined point mutation in the β-globin gene and therefore is an optimal target for hematopoietic stem cell (HSC) gene-addition/editing therapy. In HSC gene-addition therapy, a therapeutic β-globin gene is integrated into patient HSCs via lentiviral transduction, resulting in long-term phenotypic correction. State-of-the-art gene-editing technology has made it possible to repair the β-globin mutation in patient HSCs or target genetic loci associated with reactivation of endogenous γ-globin expression. With both approaches showing signs of therapeutic efficacy in patients, we discuss current genetic treatments, challenges, and technical advances in this field.

Identifiants

DOI: 10.1016/j.stem.2021.01.001 PMID: 33545079
pubmed: 33545079
pii: S1934-5909(21)00001-1
doi: 10.1016/j.stem.2021.01.001
pii:
doi:

Types de publication

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

Langues

eng

Sous-ensembles de citation

IM

Pagination

191-208

Informations de copyright

Published by Elsevier Inc.

Déclaration de conflit d'intérêts

Declaration of Interests J.F.T. and N.U. patented “Lentiviral vector for treating hemoglobin disorders (WO2016039933A1)” and “Lentiviral protein delivery system for RNA-guided genome editing (WO2017059241A1).” Except for these patents, the authors declare no competing interests.

Auteurs

Claire M Drysdale (CM)

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

Tina Nassehi (T)

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

Jackson Gamer (J)

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

Morgan Yapundich (M)

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

John F Tisdale (JF)

Cellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institute (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD 20892, USA. Electronic address: johntis@mail.nih.gov.

Naoya Uchida (N)

Cellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institute (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD 20892, USA; 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. Electronic address: uchidan@nhlbi.nih.gov.

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Classifications MeSH

questionsmedicales.fr › Phénomènes génétiques › Régulation de l'expression des gènes › Épigenèse génétique › Extinction de l'expression des gènes › Systèmes CRISPR-Cas › Hematopoietic-Stem-Cell-Targeted Gene-Addition...
questionsmedicales.fr › Techniques d'investigation › Techniques génétiques › Génie génétique › Édition de gène › Hematopoietic-Stem-Cell-Targeted Gene-Addition...
questionsmedicales.fr › Procédures de chirurgie opératoire › Transplantation › Transplantation cellulaire › Transplantation de cellules souches › Transplantation de cellules souches hématopoïétiques › Hematopoietic-Stem-Cell-Targeted Gene-Addition...
questionsmedicales.fr › Systèmes sanguin et immunitaire › Système hématopoïétique › Cellules de la moelle osseuse › Cellules souches hématopoïétiques › Hematopoietic-Stem-Cell-Targeted Gene-Addition...
questionsmedicales.fr › Malformations et maladies congénitales, héréditaires et néonatales › Maladies génétiques congénitales › Hémoglobinopathies › Hematopoietic-Stem-Cell-Targeted Gene-Addition...
questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Primates › Haplorhini › Catarrhini › Hominidae › Humains › Hematopoietic-Stem-Cell-Targeted Gene-Addition...