Using CRISPR gene editing, a team from Children’s Hospital of Philadelphia (CHOP) and Penn Medicine have thwarted a lethal lung disease in an animal model in which a harmful mutation causes... Read more
CHOP/Penn Medicine Team Shows first example of base-editing tool to treat a disease in utero in animal models For the first time, scientists have performed prenatal gene editing to prevent a... Read more
Social and ethical issues arise beyond potential for preventing rare complex diseases Sophisticated prenatal techniques, not yet in clinical practice, offer the potential to prevent a cruel... Read more
Surprisingly rapid regrowth of unused brain connections after decades of near blindness Since 2007, clinical trials using gene therapy have resulted in often-dramatic sight restoration for d... Read more
An experimental therapy has brought prolonged remissions to a high proportion of patients who were facing death from advanced leukemia after standard treatments had failed, researchers are r... Read more
New research in The FASEB Journal suggests that with magnetic guidance, specially designed nanoparticles can help deliver genes to injured arteries, using stents as the delivery platform Ste... Read more
This had never before been achieved with any other innovative therapies for diabetes. Researchers from the Universitat Autònoma de Barcelona (UAB), led by Fàtima Bosch, have shown for the fi... Read more