A gene therapy designed to replace a missing brain protein restored normal brain activity and improved behavior in a mouse ...

Gene therapy represents one of medicine’s most ambitious attempts to treat disease at its root cause by altering the genetic code itself. The approach works by recovering the functions of critical ...

Sarepta Therapeutics faces a challenging outlook, with 2025 marked by safety setbacks, missed endpoints, and a sharp price ...

Emerging gene editing platforms are revolutionizing precision medicine, enabling safe and effective genetic modifications for ...

Researchers at the Nora Eccles Harrison Cardiovascular Research and Training Institute, University of Utah, and the University of Utah School of Medicine, have demonstrated that a gene therapy can ...

A cutting-edge procedure for treating sickle cell disease is changing lives in the St. Louis area, where an estimated 1,200 ...

Automation, decentralized manufacturing, and supportive regulations are helping to surmount technical difficulties in cell and gene therapy manufacturing.

Viral vectors dominate gene therapy, with lentivirus, adenovirus, and AAV being key players, each with unique advantages and limitations. Non-viral vectors, such as lipid nanoparticles and GalNAc, ...

Dr. Roni Baha Mai and his team developed a groundbreaking neurosurgical procedure to treat a devastating genetic disorder, ...