CRISPR gene therapy Casgevy can nearly eliminate sickle cell crises, but difficult stem‑cell collection is delaying access ...

This story is part of a series on the current progression in Regenerative Medicine. In 1999, I defined regenerative medicine as the collection of interventions that restore tissues and organs damaged ...

After teasing a new regulatory process for personalized genetic medicines at the end of last year, the FDA today unveiled draft guidance for an approval pathway that could see custom CRISPR therapies, ...

Despite innovative new research, many cell and gene therapies do not make it all the way to the patients. Researchers and ...

Scientists looked at multiple techniques used to measure the modified viruses deployed in some gene therapy research and treatments. One technique, known as SEC-MALS, was the most precise and accurate ...

While gene therapy for cystic fibrosis is still in the research phase, researchers aim to correct the defective gene responsible for the disease. Current research is promising. Cystic fibrosis (CF) is ...

UCLA researchers use lipid nanoparticles to insert full CFTR gene into airway cells, restoring function and opening new ...

SEATTLE — Some of our most challenging health conditions result from genetic disorders like sickle cell disease and certain cancers. Gene therapy is revolutionizing how they're treated, and the Fred ...

For patients with rare and devastating neurological disorders, treatment options can seem few and far between. However, gene therapy research, led by Dr. Russell Lonser and Dr. Brad Elder, is hoping ...

Over the last four years, Shriners Children’s St. Louis researchers have been working to develop a new way to prevent the effects of childhood obesity. Now, using gene therapy, Shriners Children’s St.

A Yale research team has created a new computer tool that can pinpoint when exactly genes turn on and off over time during brain development - a finding that may one day help doctors identify the ...

Researchers from Tokyo Metropolitan University have created a new molecule which carries DNA into biological cells, to treat ...