Hematopoietic Stem Cell Gene Therapy: Progress and Lessons Learned

Richard A Morgan; David Gray; Anastasia Lomova; Donald B Kohn

doi:10.1016/j.stem.2017.10.010

Hematopoietic Stem Cell Gene Therapy: Progress and Lessons Learned

Cell Stem Cell. 2017 Nov 2;21(5):574-590. doi: 10.1016/j.stem.2017.10.010.

Authors

Richard A Morgan¹, David Gray², Anastasia Lomova³, Donald B Kohn⁴

Affiliations

¹ Charles R. Drew University of Medicine and Science, Los Angeles, CA, 90059; Department of Molecular and Medical Pharmacology, David Geffen School of Medicine at University of California, Los Angeles, CA, 90095.
² Molecular Biology Institute Interdepartmental Doctoral Program, University of California, Los Angeles, CA, 90095.
³ Department of Molecular and Medical Pharmacology, David Geffen School of Medicine at University of California, Los Angeles, CA, 90095.
⁴ Department of Molecular and Medical Pharmacology, David Geffen School of Medicine at University of California, Los Angeles, CA, 90095; Department of Microbiology, Immunology & Molecular Genetics, David Geffen School of Medicine at University of California, Los Angeles, CA, 90095; Department of Pediatrics, David Geffen School of Medicine at University of California, Los Angeles, CA, 90095; The Eli & Edythe Broad Center of Regenerative Medicine & Stem Cell Research, University of California, Los Angeles, CA, USA. Electronic address: [email protected].

Abstract

The use of allogeneic hematopoietic stem cells (HSCs) to treat genetic blood cell diseases has become a clinical standard but is limited by the availability of suitable matched donors and potential immunologic complications. Gene therapy using autologous HSCs should avoid these limitations and thus may be safer. Progressive improvements in techniques for genetic correction of HSCs, by either vector gene addition or gene editing, are facilitating successful treatments for an increasing number of diseases. We highlight the progress, successes, and remaining challenges toward the development of HSC gene therapies and discuss lessons they provide for the development of future clinical stem cell therapies.

Publication types

Review

MeSH terms

Clinical Trials as Topic
Gene Editing
Gene Transfer Techniques
Genetic Therapy*
Genetic Vectors / metabolism
Hematopoietic Stem Cell Transplantation / adverse effects
Hematopoietic Stem Cells / cytology*
Humans

Abstract

Publication types

MeSH terms

Grants and funding