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 muscular dystrophy, ...
Tag: gene
First Successful Gene Therapy for Huntington’s Disease Slows Disease Progression by 75%
An experimental gene therapy developed by uniQure has, for the first time, shown a dramatic slowing of Huntington's disease progression ...
Shared gene signatures reveal metabolic dysfunction in liver cirrhosis and acute-on-chronic liver failure
Background and objectives Chronic liver cirrhosis (LC) and acute-on-chronic liver failure (ACLF) are interconnected hepatic disorders associated with substantial morbidity ...
Dr. Michael Piepkorn on How the Discovery of the p16 Gene Transformed Familial Melanoma Diagnosis
The discovery of the p16 gene was a significant turning point in understanding familial melanoma. This development was primarily responsible ...
Large-scale gene analysis identifies new biomarkers and drug targets for lung adenocarcinoma
Graphical abstract. Credit: Cancer Cell (2025). DOI: 10.1016/j.ccell.2025.07.011 By analyzing tens of thousands of genes, proteins, and protein modifications in ...
New research identifies critical gene for treatment
Amyotrophic lateral sclerosis (ALS) - which you may know as the disease that affected Stephen Hawking - is a fatal ...
Ancient viral DNA found to regulate human gene expression
A new international study suggests that ancient viral DNA embedded in our genome, which were long dismissed as genetic "junk", ...
Sarepta gene therapy deaths must not halt Duchenne progress
They say death is one of life’s few certainties. For a boy or young man living with Duchenne muscular dystrophy, ...
Gene essential for vitamin D absorption could help unlock treatments for cancer and autoimmune diseases
Credit: Pixabay/CC0 Public Domain Vitamin D is not only an essential nutrient, but also the precursor of the hormone calcitriol, ...
Sarepta Slashes Staff, Maps Path Forward for Gene Therapy and Narrower Pipeline
Sarepta Therapeutics’ report of a second fatality this year in a patient dosed with its Duchenne muscular dystrophy gene therapy ...
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