Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease-Reference-Cited by-同舟云学术

Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease

Published:2021-06-16 Issue:598 Volume:13 Page:
ISSN:1946-6234
Container-title:Science Translational Medicine
language:en
Short-container-title:Sci. Transl. Med.

Author:

Lattanzi Annalisa¹²^ORCID,Camarena Joab¹^ORCID,Lahiri Premanjali³^ORCID,Segal Helen³,Srifa Waracharee¹,Vakulskas Christopher A.⁴,Frock Richard L.⁵^ORCID,Kenrick Josefin⁵^ORCID,Lee Ciaran⁶^ORCID,Talbott Narae³,Skowronski Jason³^ORCID,Cromer M. Kyle¹,Charlesworth Carsten T.¹^ORCID,Bak Rasmus O.⁷⁸,Mantri Sruthi¹^ORCID,Bao Gang⁹^ORCID,DiGiusto David³,Tisdale John¹⁰^ORCID,Wright J. Fraser¹²^ORCID,Bhatia Neehar³^ORCID,Roncarolo Maria Grazia¹²¹¹,Dever Daniel P.¹^ORCID,Porteus Matthew H.¹²¹¹^ORCID

Affiliation:

1. Department of Pediatrics, Stanford University, Stanford, CA 94305, USA.

2. Center for Definitive and Curative Medicine, Stanford University, Stanford, CA 94305, USA.

3. Laboratory for Cell and Gene Medicine, Stanford University, Stanford, CA 94304, USA.

4. Integrated DNA Technologies Inc., Coralville, IA 52241, USA.

5. Department of Radiation Oncology, Stanford University, Stanford, CA 94305, USA.

6. APC Microbiome Ireland, University College Cork, T12 YN60 Cork, Ireland.

7. Department of Biomedicine, Aarhus University, DK-8000 Aarhus, Denmark.

8. Aarhus Institute of Advanced Studies (AIAS), Aarhus University, DK-8000 Aarhus, Denmark.

9. Department of Bioengineering, Rice University, Houston, TX 77006, USA.

10. Molecular and Clinical Hematology Branch, NHLBI, Bethesda, MD 20814, USA.

11. Institute of Stem Cell Biology and Regenerative Medicine, Stanford University, Stanford, CA 94305, USA.

Abstract

Preclinical efficacy and safety data in mice provide support for ex vivo β-globin gene correction to treat patients with sickle cell disease.

Funder

Doris Duke Charitable Foundation

Chan Zuckerberg Initiative

NHLBI

CIRM

Publisher

American Association for the Advancement of Science (AAAS)

Subject

General Medicine

Reference77 articles.

1. National Heart Lung and Blood Institute Sickle Cell Disease (2020); available at https://www.nhlbi.nih.gov/health-topics/sickle-cell-disease.

2. A Phase 3 Randomized Trial of Voxelotor in Sickle Cell Disease

3. Crizanlizumab for the Prevention of Pain Crises in Sickle Cell Disease

4. Sickle cell disease: an international survey of results of HLA-identical sibling hematopoietic stem cell transplantation

5. Effect of donor type and conditioning regimen intensity on allogeneic transplantation outcomes in patients with sickle cell disease: a retrospective multicentre, cohort study

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