Read all about gene therapy including current medical research on switching therapeutic genes on and off, light-activated gene therapy and gene silencing.
A change in just one letter in the code that makes up a cancer-causing gene can significantly affect how aggressive a tumor is or how well a patient with cancer responds to a particular therapy. A new, very precise gene-editing tool will enable scientists to study the impact of these specific genetic changes in preclinical models rather than being limited to more broadly targeted tactics, such as deleting the entire gene.
New research has successfully reversed hearing loss in mice. Scientists used a genetic approach to fix deafness in mice, restoring their hearing abilities in low and middle frequency ranges.
In a step forward in the development of genetic medicines, researchers have developed a proof-of-concept model for delivering gene editing tools to treat blood disorders, allowing for the modification of diseased blood cells directly within the body. If translated into the clinic, this approach could expand access and reduce the cost of gene therapies for blood disorders, many of which currently require patients receive chemotherapy and a stem cell transplant.
