Novel Gene Therapy Vehicle Successfully Crosses Blood-Brain Barrier in Mice
Researchers at the Broad Institute have developed a groundbreaking gene therapy delivery vehicle that effectively crosses the blood-brain barrier in mice, offering new possibilities for treating a range of brain diseases. This innovative approach utilizes a human protein to transport therapeutic genes into the brain, promising safer and more efficient treatments for neurodevelopmental and neurodegenerative disorders.
Scientists Conduct First-Ever Gene Therapy Tests in Whole Human Liver
Groundbreaking study by Children’s Medical Research Institute (CMRI) conducts first-ever gene therapy tests in whole human liver, aiming to develop more effective treatments for inherited diseases. Research addresses challenges in bringing gene therapies from lab to clinic, utilizing innovative normothermic liver perfusion system to test AAV-based therapeutics before clinical studies. Senior author expresses excitement about potential for directly assessing gene therapeutics in human liver.