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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New discovery enables gene therapy for muscular dystrophies, other disorders

$1.8 million awarded to study the durability of gene therapy - EurekAlert

Gene Therapy Transforms Harmful Fats Into Beneficial Omega-3s

Gene therapy shows long-term benefit for patients with a rare pediatric brain disease

Gene therapy effective in hereditary blindness

Gene therapy effective in hereditary blindness - EurekAlert

Vision 10,000 Times Better: Gene Therapy Delivers Life-Changing Results

Gene therapy gets a turbo boost

Gene therapy gets a turbo boost from University of Hawaii researchers - EurekAlert

Machine learning approach helps researchers design better gene-delivery vehicles for gene therapy

Novel nanosensing technique for quality control of viral vectors in gene therapy

Novel nanosensing technique for quality control of viral vectors in gene therapy - EurekAlert

Review paper unveils the potential of suppressor tRNA in gene therapy - EurekAlert

Gene therapy could prevent the tau tangles linked with Alzheimer's

Gene therapy halts progression of rare genetic condition in young boy - EurekAlert

Top FDA official overrules staff to approve gene therapy that failed trial

Promising gene therapy for FOXG1 syndrome

Study suggests promising gene therapy for FOXG1 syndrome - EurekAlert

Gene therapy enables five children who were born deaf to hear

New, modified CRISPR protein can fit inside virus used for gene therapy - EurekAlert