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Science. 2018 Jan 12;359(6372). pii: eaan4672. doi: 10.1126/science.aan4672.

Gene therapy comes of age.

Author information

1
Hematology Branch, National Heart, Lung and Blood Institute, Bethesda, MD, USA. dunbarc@nhlbi.nih.gov m-sadelain@ski.mskcc.org.
2
Spark Therapeutics, Philadelphia, PA, USA.
3
Massachusetts General Hospital and Harvard Medical School, Boston, MA, USA.
4
David Geffen School of Medicine, University of California, Los Angeles, CA, USA.
5
The Institute of Medical Science, The University of Tokyo, Tokyo, Japan.
6
Memorial Sloan Kettering Cancer Center, New York, NY, USA. dunbarc@nhlbi.nih.gov m-sadelain@ski.mskcc.org.

Abstract

After almost 30 years of promise tempered by setbacks, gene therapies are rapidly becoming a critical component of the therapeutic armamentarium for a variety of inherited and acquired human diseases. Gene therapies for inherited immune disorders, hemophilia, eye and neurodegenerative disorders, and lymphoid cancers recently progressed to approved drug status in the United States and Europe, or are anticipated to receive approval in the near future. In this Review, we discuss milestones in the development of gene therapies, focusing on direct in vivo administration of viral vectors and adoptive transfer of genetically engineered T cells or hematopoietic stem cells. We also discuss emerging genome editing technologies that should further advance the scope and efficacy of gene therapy approaches.

PMID:
29326244
DOI:
10.1126/science.aan4672
[Indexed for MEDLINE]

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