Patients with a rare genetic skin condition have been successfully treated, for the first time, using a novel gene therapy...
Gene therapy given to children affected by the fatal neurological disorder Tay-Sachs disease is safe and stabilises disease progression, reports an exploratory proof-of-concept study...
In his loud shirt, covered in green leaves and plants, the University of Manchester's professor of Zoology, Matthew Cobb, was dressed appropriately for this evening's talk...
Two patients who received CAR-T gene therapy over a decade ago are still in remission, the longest follow-up study of its kind has shown...
A hidden component of the CRISPR/Cas3 genome editing system, which is essential for it to work, has been discovered by scientists...
A new gene therapy approach has shown promise in a preclinical mouse model for treating limb-girdle muscular dystrophy 2B, a rare genetic disorder which causes severe muscle weakness...
The determination of how 'serious' a condition is has direct and important implications for policy and clinical translation of existing and emerging genomic technologies...
A new technique to turn therapeutic genes on in specific cells has been developed, potentially changing the future of gene therapies...
Gene therapy delivered to a novel mouse model of Parkinson's disease improved the effects of existing Parkinson's drug levodopa...
The National Institutes for Health, Food and Drug Administration and 15 pharmaceuticals and non-profit organisations have joined together to accelerate the development of gene therapies in the USA...
