Penn, CHOP will test gene therapy for rare diseases with new FDA trial protocol

Penn and CHOP researchers want to develop urea cycle disorder treatments using CRISPR gene-editing therapy. Get unlimited access to Inquirer.com and The Inquirer App, plus 5 articles each month to ...
Nature

CRISPR vs. cholesterol: can gene editing prevent heart disease?

Promising results from a small clinical trial highlight a growing interest in designing gene-editing treatments for common ...
Precision Medicine Online

FDA Introduces 'Plausible Mechanism Pathway' to Spur Personalized Therapy Development

A sponsor will have to demonstrate success with "with several consecutive patients with different bespoke therapies" to ...
Fierce Biotech

UPDATED: Prasad, Makary unveil new FDA path for accelerating custom gene editing therapies

In a highly anticipated article, FDA officials Vinay Prasad, M.D., and Martin Makary, M.D., outlined a novel regulatory ...
Interesting Engineering on MSN

Scientists achieve targeted seizure control using sound waves and gene therapy

Bioengineers at Rice University have developed a nonsurgical method to control seizure-related brain activity using sound waves and gene therapy. The approach, tested in animal models, targets only ...
Hackaday

Gene Therapy Aims To Slow Huntington’s Disease To A Crawl

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

Gene therapy for sickle cell and β-thalassemia works by disrupting three-dimensional genome structure

Scientists at St. Jude Children's Research Hospital and Northwestern University identified a previously unknown treatment ...

Scientists have edited a gene that may reduce high cholesterol permanently

With a snip of a gene, doctors may one day permanently lower dangerously high cholesterol, possibly removing the need for ...