Gene therapy comes of age
Генная терапия достигает зрелости
2018-01-12
SCID: 54.1/4n5k2gjq
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gene therapygenetically engineered T cellsgenome editinghematopoietic stem cellsviral vectors
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Abstract (AI)
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.
Key Findings
1
Emerging genome editing technologies are expected to expand the scope and improve the efficacy of gene therapy approaches.
2
Gene therapies for inherited immune disorders, hemophilia, eye and neurodegenerative disorders, and lymphoid cancers have recently achieved regulatory approval or are near approval in the US and Europe.
3
Gene therapies have transitioned from decades of promise to becoming a critical therapeutic option for various inherited and acquired diseases.
4
The review highlights two main delivery approaches: direct in vivo administration of viral vectors and adoptive transfer of genetically engineered T cells or hematopoietic stem cells.
Research Object
Gene therapy interventions (including direct in vivo viral vector administration and adoptive transfer of genetically engineered T cells or hematopoietic stem cells)
Research Subject
Developmental milestones, clinical translation, and expanding scope and efficacy of gene therapy approaches, including approvals, in vivo viral vector delivery, adoptive cell transfer, and emerging genome editing technologies
Publication Details
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2018-01-12
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