Current Clinical Applications of In Vivo Gene Therapy with AAVs
Nationwide Children's Hospital · The Ohio State University · +10 more institutions
Abstract
Hereditary diseases are caused by mutations in genes, and more than 7,000 rare diseases affect over 30 million Americans. For more than 30 years, hundreds of researchers have maintained that genetic modifications would provide effective treatments for many inherited human diseases, offering durable and possibly curative clinical benefit with a single treatment. This review is limited to gene therapy using adeno-associated virus (AAV) because the gene delivered by this vector does not integrate into the patient genome and has a low immunogenicity. There are now five treatments approved for commercialization and currently available, i.e., Luxturna, Zolgensma, the two chimeric antigen receptor T cell (CAR-T)…
Citation impact
- FWCI
- 66.42
- Percentile
- 100%
- References
- 259
Authors
13- JRJerry R. Mendell
Nationwide Children's Hospital, The Ohio State University
- SASamiah Al-Zaidy
- LRLouise R. Rodino‐Klapac
Sarepta Therapeutics (United States)
- KGKimberly Goodspeed
Southwestern Medical Center, Southwestern Medical Center, The University of Texas Southwestern Medical Center
- SJSteven J. Gray
Southwestern Medical Center, Southwestern Medical Center, The University of Texas Southwestern Medical Center
Topics & keywords
- Genetic enhancement
- Medicine
- Choroideremia
- Spinal muscular atrophy
- Bioinformatics
- Disease
- Pathology
- Biology