A new skin-applied gene therapy uses lipid nanoparticles to deliver gene editing into skin stem cells, correcting ...

Dr. Rebecca Ahrens-Nicklas and Dr. Kiran Musunru crafted a bespoke treatment that has successfully corrected the genetic defect.

A new CRISPR-based one-off procedure that lowers "bad" cholesterol has been approved to enter Phase I human trial. If ...

Bionano Genomics, Inc. (Nasdaq: BNGO) announced a peer-reviewed study published in Molecular Therapy - Methods & Clinical Development, describing the application of optical genome mapping (OGM) to ...

Stanford researchers and their collaborators have revealed a new device that could change the way scientists conduct gene-editing experiments. The device, CRISPR-GPT, is an artificial intelligence lab ...

The hold followed Intellia’s voluntary pause in response to a grade 4 liver event—a case of Hy’s Law—in the ATTR-CM trial.

Intellia is instituting new safeguards following the death of a study participant that led regulators to pause two trials in people with transthyretin amyloidosis. One of those tests remains suspended ...

CHICAGO (Reuters) -A single infusion of CRISPR Therapeutics' experimental gene therapy was safe and reduced levels of harmful LDL cholesterol and triglycerides by half in four people taking the ...

Delivery of therapeutic genes is essential for gene therapy. Adeno-associated viruses (AAVs) are a prime vector for carrying ...

Gene therapy is a medical treatment that works by changing or fixing a person's genes to treat or prevent disease ...

Developing a gene therapy typically takes years, but when Baby KJ was diagnosed with a deadly genetic condition, scientists had only months. What followed was a successful collaboration among ...