Gene therapy comes of age Journal Article
| Authors: | Dunbar, C. E.; High, K. A.; Joung, J. K.; Kohn, D. B.; Ozawa, K.; Sadelain, M. |
| Article Title: | Gene therapy comes of age |
| 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. |
| Keywords: | unclassified drug; review; nonhuman; united states; t lymphocyte; gene; immune system; europe; genetic engineering; gene therapy; lentivirus vector; retrovirus vector; adoptive transfer; genome; hematopoietic stem cell; eye disease; genetic disorder; blindness; severe combined immunodeficiency; immunopathology; degenerative disease; heritability; virus vector; hemophilia; disease; cell; lymphatic system disease; drug; aromatic levo amino acid decarboxylase; spinal muscular atrophy; human; priority journal; gene editing; neurturin; adeno associated virus vector; alipogene tiparvovec; nusinersen; retinal pigment epithelium 65; spumavirus vector; strimvelis; visual pigment; hyperlipoproteinemia type 1 |
| Journal Title: | Science |
| Volume: | 359 |
| Issue: | 6372 |
| ISSN: | 0036-8075 |
| Publisher: | American Association for the Advancement of Science |
| Date Published: | 2018-01-12 |
| Start Page: | eaan4672 |
| Language: | English |
| DOI: | 10.1126/science.aan4672 |
| PROVIDER: | scopus |
| PUBMED: | 29326244 |
| DOI/URL: | |
| Notes: | Review -- Export Date: 1 February 2018 -- Source: Scopus |
