Gene therapy is any effort to alter the roughly 20,000 protein-encoding genes in the human body to improve its function and produce the proteins it needs. Single-gene mutations can be harmless, but they may also result in serious or life-threateningconditions, including cystic fibrosis, Tay-Sachs disease, and muscular dystrophies.
> Deaf kids and adults are hearing for the first time with a novel gene therapy. (More)
> Prenatal gene therapy is eliciting interest with a planned clinical trial. (More)
Gene therapies aim to correct errors in variant genes by replacing them with healthy copies that were created in the lab. These genes can be delivered into the body using a virus emptied of its disease-causing instructions and instead carrying functional genes. Inside the body, the virus does what it typically does—invades the patient’s cells—but instead of making someone sick, it delivers healthy genes so the body has the instructions it needs to produce the proteins it needs, shut off protein production, or perform other necessary actions.
> See how viruses act as delivery vehicles for the new DNA. (More)
> Gene therapy can also be conducted outside the body. (More)
> There’s been some recent progress in animals regarding a rare genetic disorder that can cause people to become deaf and blind. (More)
Gene-editing technologies like CRISPR have enabled breakthrough treatment options in recent years. In the US, there are currently approved gene therapies for conditions including sickle cell disease, Duchenne muscular dystrophy, leukemia and lymphoma, inherited retinal and hearing loss disorders, and more.
> Explore this list of more than 12 FDA-approved gene therapies.
> The FDA has recently proposed a way to speed reviews for gene therapy approvals without lengthy clinical trials. (More)
> Retinal cells have recently been regenerated in the lab with gene therapy. (More)
Discover more:
> Elizabeth Taylor had two rows of eyelashes due to a gene mutation. (More)
> Base editing might be CRISPR 2.0. (More)
> On an emergency basis, a baby with a toxic ammonia buildup disorder was successfully treated with a bespoke gene therapy. (More)
>… explore everything we’ve learned about Gene Therapy.
