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. In 2003, Gendicine became the first gene therapy to receive regulatory approval.

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Promising gene therapy delivers treatment directly to brain

Making progress with a gene therapy for muscular dystrophy

Infant gene therapy is a breakthrough for Artemis-SCID patients

Gene therapy restored immune system in children with rare disorder

Gene Therapy: A New Age

'Butterfly disease' makes the skin incredibly fragile, but a new gene therapy helps it heal

Gene therapy for heart attacks in mice just got more precise

$3.5-Million Hemophilia Gene Therapy Is World's Most Expensive Drug

New gene therapy restores night vision of people with inherited eye disorder

Gene Therapy Can Restore Night Vision After Decades of Congenital Blindness

This Gene Therapy Could Keep Calm The Overactive Neurons That Cause Epileptic Seizures

Gene therapy targeting overactive brain cells could treat neurological disorders

Proof-of-concept study advances potential new way to deliver gene therapy

Gene therapy rapidly improves night vision in adults with congenital blindness

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

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