Celia Grace Hamlett was 4-years-old when she traveled with her parents from Alabama to Minneapolis to receive life-saving treatment for a rare genetic disease, Metachromatic Leukodystrophy or MLD.

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 ...

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 is a revolutionary field in modern medicine, offering transformative potential to treat and potentially cure a wide range of genetic and acquired diseases. By addressing the root cause of ...

A new gene therapy can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the amount of blood the heart can pump and dramatically improves ...

The search for gene therapies to treat rheumatoid arthritis (RA) has moved in fits and starts over the past two decades, with trials starting and stopping, and their focus shifting to osteoarthritis ...

News broke yesterday that researchers in Philadelphia appear to have successfully treated a 6-month-old baby boy, called KJ, with a personalized CRISPR gene-editing therapy. The treatment corrects an ...

Vence L. Bonham Jr., Acting Deputy Director at the National Human Genome Research Institute (NHGRI) and an investigator in the National Human Genome Research Institute’s (NHGRI) Division of Intramural ...

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 ...