Breakthrough Gene Therapy Helps Blind Patients Regain Partial Vision, Offering New Hope for Millions
Scientists have achieved a promising breakthrough in the treatment of blindness, demonstrating that an experimental gene therapy can partially restore vision in people who have lost their sight because of a severe inherited eye disease. The findings, published in the New England Journal of Medicine, represent an important advance in regenerative medicine and suggest that […]
One-Time Gene Therapy Shows Lasting Promise Against Huntington’s Disease Four Years Later
An experimental one-time gene therapy from biotech company uniQure continues to show signs of slowing Huntington’s disease four years after treatment, offering potentially important evidence that gene therapy could alter the course of a devastating inherited neurological disorder for which no approved disease-modifying treatment currently exists. The treatment, called ifezuntirgene inilparvovec, or AMT-130, is designed […]
One CRISPR Infusion Cuts Dangerous Cholesterol for a Year in Early Human Trial
An experimental gene-editing therapy has produced substantial and lasting reductions in dangerous blood fats after only a single treatment, offering early evidence that CRISPR technology could eventually transform how some severe cholesterol disorders are treated. Researchers from the Cleveland Clinic reported that a one-time infusion of the experimental therapy CTX310 reduced LDL cholesterol — commonly […]
