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 ...

In a first for adults in Singapore, scientists are conducting gene editing trials on heart patients to correct defects at their genetic source.

From revolutionary CRISPR treatment to heavy drinking dangers, here's what you need to know about the latest health research.

Earlier this year, researchers at CHOP and Penn created a first-of-its-kind drug customized to a unique genetic mutation to save an infant named Baby KJ from dying of a rare liver disorder. KJ Muldoon ...

The approach is “very exciting,” but one expert says a lot more research is needed, especially around safety, before clinical ...

Researchers report early but encouraging results on an experimental gene-editing treatment for high cholesterol.

In a 15-patient, Phase 1, first-in-human trial, a one-time CRISPR-Cas9 gene-editing therapy safely reduced LDL cholesterol ...

Fractyl Health, Inc. announced promising preclinical results for its gene therapy RJVA-001 at the 2025 ASGCT Annual Meeting, showing that a single dose leads to substantial metabolic improvements ...

ImmunoAct, backed by government funding, is advancing CAR-T cell therapy to make cancer treatment more accessible. Their product, NexCAR19, uses patients' immune systems to fight cancer. With support ...