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

The malignant pediatric brain tumor medulloblastoma is driven by proteins deep within the cancer cells, for which there are ...

Fred Hutch bioengineer Dr. Matthias Stephan is working to develop a special foam that could help make gene therapy for blood ...

In particular, adeno-associated virus (AAV) vectors have recently garnered significant attention from the scientific community, given their potential to serve as nucleic acid vaccines for diseases ...

Scientists discover a new gene therapy technique that may reverse ALS by restoring vital RNA signals, providing hope for ...

Despite the best efforts of modern medicine, Huntington’s disease is a condition that still comes with a tragic prognosis.

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 ...

Scientists looked at multiple techniques used to measure the modified viruses deployed in some gene therapy research and treatments. One technique, known as SEC-MALS, was the most precise and accurate ...

So, what are retrons? They’re bacterial immune systems that save the bacterial herd by culling individual bacteria infected ...

A team led by Mass Eye and Ear researchers has demonstrated for the first time a successful restoration of hearing through a novel, in vivo genome editing approach in an adult mouse model with a form ...

April 17, 2019 • The latest advance is not only encouraging news for patients with severe combined immunodeficiency. It's a test case for all those scientists working to develop better gene therapy ...