Open AccessGene Therapy 2017: Progress and Future Directions
Author(s) -
Keeler AM,
ElMallah MK,
Flotte TR
Publication year - 2017
Publication title -
clinical and translational science
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.303
H-Index - 44
eISSN - 1752-8062
pISSN - 1752-8054
DOI - 10.1111/cts.12466
Subject(s) - genetic enhancement , gene delivery , nucleic acid , transfection , vectors in gene therapy , dna , endocytosis , biology , cationic liposome , viral vector , liposome , vector (molecular biology) , chemistry , microbiology and biotechnology , biochemistry , recombinant dna , gene , cell
Gene therapy has changed dramatically in the 28 years since the first human gene transfer experiment in 1989. Alipogene tiparvovec, GlyberaR R ©, a recombinant adenoassociated virus (rAAV) product for lipoprotein lipase deficiency, and Strimvelis R ©, a lentivirus vector for severe combined immune deficiency are approved in Europe. An rAAV2 product for a congenital form of blindness is currently under review in the United States, likely to be followed by numerous other gene therapies.