With promise to slow aging and cure some types of cancer, a new gene therapy presents cost and access hurdles for CT patients ...

A TOPOGRAPH-based hierarchy stratifies biomarker–drug pairs from regulatory/positive trial evidence (tiers 1–3A) to repurposing across histotypes (3B), preclinical/early data (4), and suspected ...

Mayo Clinic researchers used milk-based nanoparticles to deliver siRNA into bile duct cancer cells. This targeted therapy ...

UAB is participating in a Phase I/II trial of AMT 191, a one time gene therapy designed to enable patients with classic Fabry disease to produce their own missing enzyme, potentially reducing the need ...

Scientists have developed a method to reprogram cancer-fighting immune cells directly inside the body, potentially eliminating the need for complex lab-based manufacturing. Early results show rapid ...

OCGN's gene-agnostic eye therapy aims to treat multiple retinal mutations, with key filings and pivotal data in 2026???2027 set to test its broad platform.

Until recently, even the most advanced gene therapies could only be given after a child was born—often racing against time to prevent irreversible damage. In the first part of this series, we explored ...

Nemours Children's Hospital is now offering Lyfgenia gene therapy. The treatment aims to reduce pain crises and hospital visits for young patients.

Researchers at Memorial Sloan Kettering Cancer Center (MSK) have made an important discovery about how genetic mutations in ...

Scott Pelley, one of the most experienced and awarded journalists today, has been reporting stories for 60 Minutes since 2004. The 2024-25 season is his 21st on the broadcast. Scott has won half of ...

Hormone therapy is widely used to treat menopausal symptoms such as hot flashes and night sweats. But scientists have long ...