Researchers have unveiled a way to flip genes back on without slicing into the genome, a shift that could make CRISPR far ...

Decades ago, researchers showed that Down syndrome (also known as trisomy 21), was caused when individuals carried an extra copy of chromosome 21. Down syndrome is also the most common genetic cause ...

A new gene therapy technique may be used to treat imflammatory bowel disease and to relieve pain and inflammation. Aditi Bhargava, PhD, associate professor at the University of California San ...

A new study has brought scientists one step further in the direction of developing a cure for a brutal group of rare brain disorders known as SYNGAP1-related disorders, or SRDs. Researchers were able ...

In a finding with implications for one of the most promising cutting-edge medical treatments, researchers evaluated several measurement techniques commonly used in gene therapy. The study determined ...

For more than a decade, the strongest AIDS drugs could not fully control Matt Chappell's HIV infection. Now his body controls it by itself, and researchers are trying to perfect the gene editing that ...

Boston biotech Beam Therapeutics has announced initial data from a phase 1/2 trial of its gene therapy for a genetic lung and liver disease, with no serious adverse events reported among the nine ...

US scientists discover a new gene therapy technique that is able to modify prostate cancer cells so that a patient's body attacks and kills them.

Gene therapy is a revolutionary field in modern medicine, offering transformative potential to treat and potentially cure a wide range of genetic and acquired diseases. By addressing the root cause of ...

A brain-machine interface company has investigated the problems found in today's intracranial implants and come to a game-changing conclusion: If the mountain won't come to them, then they'll have to ...

A revolutionary gene-silencing technique has shown that a single treatment can dramatically lower LDL cholesterol levels for over a year—without permanently altering DNA. Could this be the future of ...