The gene-editing technique known as CRISPR is promising to revolutionize medicine. Some researchers are trying to help make it available for people with very rare genetic disorders. A gene-editing ...

Cutting-edge gene editing technology could eradicate Down syndrome, according to Japanese scientists. Down syndrome, which causes a range of developmental differences and affects 1 in 700 newborns in ...

For some diseases, gene therapies offer the potential for lifelong disease amelioration and even cure. And these immensely important novel biotechnologies may be on the cusp of a boom. That is in part ...

Gene editing techniques may eventually allow trisomy to be treated at the cellular level, according to an in vitro proof-of-concept study. Down syndrome is caused by the presence of a third copy of ...

Dr. Musunuru and Dr. Ahrens-Nicklas holding KJ post infusion Senior Correspondent Gene therapy has always held enormous promise to correct genetic diseases, but turning that potential into treatments ...

Advances in the gene-editing technology known as CRISPR-Cas9 over the past 15 years have yielded important new insights into the roles that specific genes play in many diseases. But to date this ...

Vertex Pharmaceuticals (NASDAQ: VRTX) has made it to the major leagues in the world of biotech. The company is the leading maker of cystic fibrosis (CF) treatments, with its products generating more ...

These horses might look like ordinary horses, but there is something highly unusual about their genomes. They are the first of their species to have their DNA edited using CRISPR–Cas9, a technique ...

Crispr Therapeutics, a leader in CRISPR/Cas9 biotechnology, recently received FDA and EMA approval for Casgevy, the first CRISPR/Cas9-based treatment for TDT and SCD. The partnership with Vertex ...