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teasing a new regulatory process for personalized genetic medicines at the end of last year, the FDA today unveiled draft guidance for an approval pathway that could see custom CRISPR therapies, ...

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 ...

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

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 ...

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

Analyzing the purity of adeno-associated virus (AAV)-based gene therapy products requires effective methods for detecting host proteins, DNA, and incomplete viral particles in samples. It is a ...

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 ...

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.

The UAE has launched two gene therapy projects targeting the root causes of autism and rare brain disorders, marking a significant shift from symptom management to biological repair. Collaborating ...

Some gene therapy treatments use modified adeno-associated viruses (AAVs) like the one shown here to deliver therapeutic genetic material into a patient’s cells. In a finding with implications for one ...