Review
doi: 10.1093/abbs/gms036.
Epub 2012 May 23.
Phoenix rising: gene therapy makes a comeback
Affiliations
- PMID: 22623503
- PMCID: PMC7109785
- DOI: 10.1093/abbs/gms036
Item in Clipboard
Review
Phoenix rising: gene therapy makes a comeback
Acta Biochim Biophys Sin (Shanghai).
2012 Aug.
Abstract
Despite the first application of gene therapy in 1990, gene therapy has until recently failed to meet the huge expectations set forth by researchers, clinicians, and patients, thus dampening enthusiasm for an imminent cure for many life-threatening genetic diseases. Nonetheless, in recent years we have witnessed a strong comeback for gene therapy, with clinical successes in young and adult subjects suffering from inherited forms of blindness or from X-linked severe combined immunodeficiency disease. In this review, various gene therapy vectors progressing into clinical development and pivotal advances in gene therapy trials will be discussed.
References
-
- Watson JD, Crick FH. The structure of DNA. Cold Spring Harb Symp Quant Biol. 1953;18:123–131. - PubMed
-
- Keeler CE. Gene therapy. J Hered. 1947;38:294–298. - PubMed
-
- Friedmann T, Roblin R. Gene therapy for human genetic disease? Science. 1972;175:949–955. - PubMed
-
- Blaese RM, Culver KW, Chang L, Anderson WF, Mullen C, Nienhuis A, Carter C, et al. Treatment of severe combined immunodeficiency disease (SCID) due to adenosine deaminase deficiency with CD34+ selected autologous peripheral blood cells transduced with a human ADA gene. Amendment to clinical research project, Project 90-C-195, January 10, 1992. Hum Gene Ther. 1993;4:521–527. - PubMed
-
- Blaese RM, Culver KW, Miller AD, Carter CS, Fleisher T, Clerici M, Shearer G, et al. T lymphocyte-directed gene therapy for ADA-SCID: initial trial results after 4 years. Science. 1995;270:475–480. - PubMed
