Gene Therapy

Gene therapy is a medical field which focuses on the genetic modification of cells to produce a therapeutic effect or the treatment of disease by repairing or reconstructing defective genetic material. The first attempt at modifying human DNA was performed in 1980, by Martin Cline, but the first successful nuclear gene transfer in humans, approved by the National Institutes of Health, 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. It is thought to be able to cure many genetic disorders or treat them over time. Between 1989 and December 2018, over 2,900 clinical trials were conducted, with more than half of them in phase I. As of 2017, Spark Therapeutics' Luxturna and Novartis' Kymriah are the FDA's first approved gene therapies to enter the market.

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Gene therapy infused into the brain eases rare condition in children

Researchers identify a gene therapy target for polycystic kidney disease

Gene Therapy Successfully Restores Cone Function in Colorblind Children

Researchers develop gene therapy for rare ciliopathy

Stem cell-gene therapy shows promise in ALS safety trial

News at a glance: New gene therapy, Europe’s drought, and a black hole’s photon ring

New gene therapy shows promise for treating eye condition affecting millions across the globe

Gene therapy partly restores cone function in two completely colorblind children

Gene therapy approach shows promise in treating ALS

Gene therapy trial shows early success in people with haemophilia B

Novel gene therapy could reduce bleeding risk for haemophilia patients

Mouse study shows gene therapy may correct creatine deficiency disorder

A Potential Cure for Autism: New Research Finds That Gene Therapy Could Treat Pitt-Hopkins Syndrome

Gene therapy for pancreatic cancer: Experimental approach shrank tumors in one patient

Gene Therapy Successfully Treats Spinal Cord Injuries Without Side Effects

Trial Shows Gene Therapy Results with Sickle Cell Disease

Gene therapy could treat Pitt-Hopkins syndrome, proof-of-concept study suggests

Gene therapy shows promise in treating neuropathy from spinal cord injuries

Gene Therapy Reverses Effects of Autism-Linked Mutation in Human Brain Organoids

Gene therapy reverses effects of autism-linked mutation in brain organoids