Gene therapy

Gene therapy

Last Reviewed : 12/25/2020
Gene therapy

Gene therapy is the therapeutic delivery of nucleic acid polymers into a patient's cells as a drug to treat disease.

The first attempt at modifying human DNA was performed in 1980 by Martin Cline, but the first successful and approved nuclear gene transfer in humans was performed in May 1989. The first therapeutic use of gene transfer as well as the first direct insertion of human DNA into the nuclear genome was performed by French Anderson in a trial starting in September 1990.

Between 1989 and February 2016, over 2,300 clinical trials had been conducted, more than half of them in phase I.

It should be noted that not all medical procedures that introduce alterations to a patient's genetic makeup can be considered gene therapy. Bone marrow transplantation and organ transplants in general have been found to introduce foreign DNA into patients. Gene therapy is defined by the precision of the procedure and the intention of direct therapeutic effects.


We researched this topic for you and found the following best online resources. They are categorized into basic, advanced, and research level based on the extent of information you need. You will be taken to the respective websites by pressing on the links below.


Basic information: genetics home reference what is gene therapy? mayo clinic overview your genome what is gene therapy? news medical what is gene therapy? better health gene therapy kids health gene therapy very well what is gene therapy?


Advanced information: science daily gene therapy medicine net gene therapy - the future is here! patient gene therapy


Research: braz j med biol res, november 2011, volume 44(11) 1097-1104 adeno-associated virus for cystic fibrosis gene therapy by s.v. Martini et al., jabar zaman khan khattak, sobiah rauf, zubair anwar, hussain mustatab wahedi and talal jamil, 2012. Recent advances in genetic engineering - a review. Current research journal of biological sciences, 4(1): 82-89. bilsland ae, spiliopoulou p and evans trj. Virotherapy: cancer gene therapy at last? [version 1; referees: 2 approved]. F1000research 2016, 5(f1000 faculty rev):2105 (doi: 10.12688/f1000research.8211.1) yu huang, xin liu, lanlan dong, zhongchun liu, xiaohua he, and wanhong liu, “development of viral vectors for gene therapy for chronic pain,” pain research and treatment, vol. 2011, article id 968218, 8 pages, 2011. Doi:10.1155/2011/968218 targeting a genetic defect: cystic fibrosis transmembrane conductance regulator modulators in cystic fibrosis by nico derichs et al., european respiratory review 2013 22: 58-65; doi: 10.1183/09059180.00008412

Bali a, bali d, sharma a. An overview of gene therapy in head and neck cancer. Indian j hum genet [serial online] 2013 [cited 2016 sep 29];19:282-90. Available from: tarja malm, jari koistinaho, and katja kanninen, “utilization of appswe/ps1de9 transgenic mice in research of alzheimer's disease: focus on gene therapy and cell-based therapy applications,” international journal of alzheimer’s disease, vol. 2011, article id 517160, 8 pages, 2011. Doi:10.4061/2011/517160 gene therapy of muscular dystrophy hum. Mol. Genet. (2002) 11 (20):2355-2362.doi: 10.1093/hmg/11.20.2355 gene therapy: progress and predictions by mary collins, adrian thrasher published 23 december 2015.doi: 10.1098/rspb.2014.3003


Other helpful resources(support groups): american society of gene and cell therapy alliance for cancer gene therapy


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Presentations/quiz/newspaper articles: the guardian gene therapy offers hope for treatment of sickle cell anaemia scientific american how does gene therapy work? wired gene therapy emerges from disgrace to be the next big thing, again washington post gene therapies offer dramatic promise but shocking costs bbc news gene therapy reverses sight loss and is long-lasting stat news gene therapy treats duchenne muscular dystrophy in mice economist why gene-therapy drugs are so expensive times of india bangalore turns to gene therapy reuters europe gives green light to first gene therapy for children cc news l.a. Gene therapy restores leamington, ont., boy's immune system news scientist gene therapy cures blindness by replacing vision cells in eyes

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