Latest Gene therapy Stories
Infants with Batten disease, a rare but fatal neurological disorder, appear healthy at birth.
Researchers at the UCLA stem cell center and the departments of chemistry and biochemistry and pathology and laboratory medicine have identified, for the first time, a generic way to correct mutations in human mitochondrial DNA by targeting corrective RNAs, a finding with implications for treating a host of mitochondrial diseases.
A newly developed, genetically modified pig may hold the keys to the development of improved treatments and possibly even a cure for retinitis pigmentosa (RP), the most common inherited retinal disease in the United States.
A new research discovery by a team of Stanford and European scientists offers hope that people with atherosclerotic disease may one day be able to avoid limb amputation related to ischemia.
A repression of gene activity in the brain appears to be an early event affecting people with Alzheimer's disease, researchers funded by the National Institutes of Health have found.
As part of a national collaboration, Oregon Health & Science University researchers are studying specially bred mice that are more like humans than ever before when it comes to genetic variation.
Gene therapy for congenital blindness has taken another step forward, as researchers further improved vision in three adult patients previously treated in one eye. After receiving the same treatment in their other eye, the patients became better able to see in dim light, and two were able to navigate obstacles in low-light situations. No adverse effects occurred.
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