Gene therapy comes of age
Top Cited Papers
Open Access
- 12 January 2018
- journal article
- review article
- Published by American Association for the Advancement of Science (AAAS) in Science
- Vol. 359 (6372), 175-+
- https://doi.org/10.1126/science.aan4672
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.Funding Information
- National Institutes of Health ((CCSG) (P30 CA008748).)
This publication has 149 references indexed in Scilit:
- Genomic Editing of the HIV-1 Coreceptor CCR5 in Adult Hematopoietic Stem and Progenitor Cells Using Zinc Finger NucleasesMolecular Therapy, 2013
- Repurposing CRISPR as an RNA-Guided Platform for Sequence-Specific Control of Gene ExpressionCell, 2013
- The AAV Vector Toolkit: Poised at the Clinical CrossroadsMolecular Therapy, 2012
- FLASH assembly of TALENs for high-throughput genome editingNature Biotechnology, 2012
- Stem cell gene therapy: the risks of insertional mutagenesis and approaches to minimize genotoxicityFrontiers of Medicine, 2011
- Chimeric Antigen Receptor–Modified T Cells in Chronic Lymphoid LeukemiaThe New England Journal of Medicine, 2011
- Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemiaNature, 2010
- Genome editing with engineered zinc finger nucleasesNature Reviews Genetics, 2010
- A Phase I Study of Aromatic L-Amino Acid Decarboxylase Gene Therapy for Parkinson's DiseaseMolecular Therapy, 2010
- Establishment of HIV-1 resistance in CD4+ T cells by genome editing using zinc-finger nucleasesNature Biotechnology, 2008