Harmful cholesterol levels cut in half with one-time gene editing drug in early trial
Altering a single gene may help people lower dangerously high levels of cholesterol and other fats in the…
TAU researchers develop groundbreaking gene therapy to treat hearing, balance disabilities
“These findings highlight the potential of self-complementary AAVs to reduce dose requirements, minimize toxicity, and broaden clinical use…
New method could improve manufacturing of gene-therapy drugs | MIT News
Some of the most expensive drugs currently in use are gene therapies to treat specific diseases, and their…
New gene-editing method can correct many disease-causing mutations in mammalian cells
Some genetic disorders-such as cystic fibrosis, hemophilia and Tay Sachs disease-involve many mutations in a person’s genome, often…
Experimental gene therapy successfully treats dozens of children with rare fatal disorder
ADVERTISEMENT An experimental gene therapy has successfully treated dozens of children with a rare and fatal immune disorder,…
Sarepta to seek approval for gene therapy in rare muscular dystrophy
An experimental gene therapy from Sarepta Therapeutics increased levels of the gene missing in an ultra-rare form of…
Highly targeted gene therapy could revolutionize treatment for cancers linked to herpesvirus
In a preclinical study, UC Davis Comprehensive Cancer Center scientists developed a highly targeted gene therapy that could…
Harnessing “DNA surgery” to develop therapeutic medicines for genetic liver and heart diseases
Genome editing is increasingly recognzed as a leading frontier in biomedical research and an essential driver of healthcare…
In Major Breakthrough, Scientists Successfully Treat Devastating Brain Disease Huntington’s for the First Time
A research team says that for the first time in history, it’s successfully treated Huntington’s disease, a devastating…
Next-generation gene therapy tools built by UH scientist
Reading time: 2 minutesAssistant Professor Jesse Owens using a program to model the structure of his integrase protein.…
Gene Therapy Tackles Huntington’s Progress in Landmark Trial. “This Changes Everything”
A complex genetic procedure offers new hope in the fight against one of the world’s cruellest diseases. The…
Gut microbial imbalance linked to chronic sickle cell disease pain
A research collaboration involving scientists and students at The University of Texas at Dallas has found a clear…