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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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

Nanoparticles deliver gene therapy to hard-to-reach brain tumors

Gene therapy relieves back pain, repairs damaged disc in mice

The future of gene therapy has arrived, and it's changing lives - EurekAlert

Gene therapy hailed as ‘medical magic wand’ for hereditary swelling disorder

Gene therapy cures hereditary deafness in two people

Gene therapy restores hearing in children with hereditary deafness

Gene Therapy Allows an 11-Year-Old Boy to Hear for the First Time

Base editing, a new form of gene therapy, sharply lowers bad cholesterol in clinical trial

Redefining Gene Therapy: CRISPR’s Innovative “Find-and-Replace” Genome Editing

New methods for effective transport of large genes in gene therapy

New Trials Aim to Restore Hearing in Deaf Children—With Gene Therapy

Researchers design gene therapy that can effectively target glioblastoma