Characterization of Gene Alterations following Editing of the β-Globin Gene Locus in Hematopoietic Stem/Progenitor Cells-Reference-Cited by-同舟云学术

Characterization of Gene Alterations following Editing of the β-Globin Gene Locus in Hematopoietic Stem/Progenitor Cells

Published:2018-02 Issue:2 Volume:26 Page:468-479
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Long Joseph,Hoban Megan D.,Cooper Aaron R.,Kaufman Michael L.,Kuo Caroline Y.,Campo-Fernandez Beatriz,Lumaquin Dianne,Hollis Roger P.,Wang Xiaoyan,Kohn Donald B.,Romero Zulema

Funder

California Institute for Regenerative Medicine

Doris Duke Charitable Foundation

Whitcome Predoctoral Training Program

UCLA Molecular Biology Institute

Initiative to Maximize Student Diversity

NIH

CSUN-UCLA Bridges to Regenerative Medicine Program

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

Reference37 articles.

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3. Correction of sickle cell disease in transgenic mouse models by gene therapy;Pawliuk;Science,2001

4. Correction of a mouse model of sickle cell disease: lentiviral/antisickling β-globin gene transduction of unmobilized, purified hematopoietic stem cells;Levasseur;Blood,2003

5. β-globin gene transfer to human bone marrow for sickle cell disease;Romero;J. Clin. Invest.,2013

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1. Non-viral DNA delivery and TALEN editing correct the sickle cell mutation in hematopoietic stem cells;Nature Communications;2024-06-11

2. Genome editing for sickle cell disease: still time to correct?;Frontiers in Pediatrics;2023-11-02

3. Gene Therapy for Hemoglobinopathies;Human Gene Therapy;2023-09-01

4. Clinical genome editing to treat sickle cell disease—A brief update;Frontiers in Medicine;2023-01-09

5. Hematopoietic Stem Cell Gene-Addition/Editing Therapy in Sickle Cell Disease;Cells;2022-06-04