CORONA, Calif. (KABC) -- A teenage girl is alive thanks to an experimental gene therapy treatment created at UCLA. She was born with a rare genetic disorder, so any common infection could've killed ...

"Regulatory flexibility must be tailored for cell and gene therapies," commented FDA Commissioner Marty Makary. "These are ...

Cell and gene therapies use the body's building blocks - cells and genetic material - to boost the immune system or correct ...

There is no cure for the rare disease Hereditary Spastic Paraplegia (HSP), but researchers from Drexel University’s College of Medicine and the UMass Chan Medical School have achieved ...

Clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated (Cas) proteins are core components of fast-evolving therapeutic gene editing tools. Scientists have used CRISPR ...

Viral vectors dominate gene therapy, with lentivirus, adenovirus, and AAV being key players, each with unique advantages and limitations. Non-viral vectors, such as lipid nanoparticles and GalNAc, ...

Cell and gene therapy are revolutionary approaches to treating diseases at the molecular level. They involve the manipulation ...

Lowering cholesterol is one of the most effective ways to reduce your risk of heart disease, and it may soon be possible to get a one-and-done gene therapy to keep cholesterol and triglyceride levels ...

As we continue our series exploring genetic medicine, both in this story and in Destiny's Child No Longer: Rewriting Genetic Fate, gene therapy stands at a crossroads. On June 16, Sarepta Therapeutics ...