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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Scientists Restore Limited Vision in Blind Patients Using Gene Therapy and Special Goggles

Gene therapy and special goggles give some blind people limited sight

Gene therapy and special goggles give some blind people limited sight

Wiskott-Aldrich Syndrome: Long-term results of gene therapy developed at the San Raffaele-Telethon Institute published in the New England Journal of Medicine

Gene therapy for deadly childhood liver disease - EurekAlert!

Gene therapy reverses Fragile X deficits in mice - EurekAlert!

Gene therapy for hereditary hearing loss: Comprehensive overview and clinical applications - EurekAlert!

Malcom Brenner to lead newly-formed Houston Methodist Center for Cell and Gene Therapy - EurekAlert!

First-in-the-world gene therapy delivers missing gene directly to infant's brain, marking historic milestone in precision medicine - EurekAlert!

Stem cell transplantation more cost-effective than gene therapy for sickle cell disease - EurekAlert!

Critical Path Institute awards $249,719 grant to Odylia Therapeutics to advance gene therapy for USH1C-associated vision loss - EurekAlert!

Boy’s brain tumor tied to gene therapy

Toloo Taghian awarded $3.2M to develop gene therapy for a rare genetic disease - EurekAlert!

Gene therapy for deafness approved

Hearing restoration from gene therapy for inherited deafness lasts years, new trial results show - EurekAlert!

‘Oscar of science’ awarded to team behind gene therapy that restores lost vision

FDA approves gene therapy for severe leukocyte adhesion deficiency-I, a rare immune disorder - EurekAlert!

Incurable muscle disease: First successes in the development of a gene therapy - EurekAlert!

Cell and gene therapy across 35 years - EurekAlert!

AAVLINK: Potent DNA-recombination method for large cargo delivery in gene therapy - EurekAlert!