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teasing a new regulatory process for personalized genetic medicines at the end of last year, the FDA today unveiled draft guidance for an approval pathway that could see custom CRISPR therapies, ...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

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

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

Non-profit organisations play a crucial role in supporting rare disease therapy development and rescuing shelved programmes, ...

Delivery of therapeutic genes is essential for gene therapy. Adeno-associated viruses (AAVs) are a prime vector for carrying gene cargoes because of their superior gene segmentation flexibility 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 ...

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 ...

Gene therapy and gene editing are marvels of modern biotechnology, providing revolutionary techniques for fixing genetic abnormalities. Gene therapy introduces functional genetic material to ...

The UAE has launched two gene therapy projects targeting the root causes of autism and rare brain disorders, marking a ...

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 ...