Browsing: gene

Researchers in Australia have developed a gene therapy that could restore heart function in children born with mutations that cause cardiomyopathy. A study published inNature Cardiovascular Researchsuggests this approach could potentially address more than 25% of cardiomyopathies that have a genetic basis. 

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Experimental evolution through selection experiments is a vital tool for exploring the special features of polygenic traits. Here, we provide liver transcriptome data from a paternally selected marathon mouse model (DUhTP), characterized by exceptional running performance, and unselected controls (DUC), both descended from the same polygenic background. To study training responses, both lines were either…

“Burn fat, build muscle.” It’s a familiar workout slogan, but the benefits go far beyond aesthetics. Having less belly fat and more muscle guards against heart attacks, Type 2 diabetes, and a host of other metabolic diseases

More than one-third of the global population has metabolic dysfunction-associated steatotic liver disease, or MASLD, a condition in which fat accumulates in the liver. MASLD can progress to more severe diseases like metabolic dysfunction-associated steatohepatitis, cirrhosis and hepatocellular carcinoma. Treatment options are limited and focus on weight loss through lifestyle changes and the use of…

The two-year treatment targeting protein production by disease-causing mutations dramatically reduced seizures and sparked developmental gains, helping one teenager walk independently

The US government’s health research moonshot agency has launched a five-year, $160 million bet that the era of bespoke gene-editing cures — currently limited to children whose families can access a vast scientific team willing to absorb the cost — can be converted into an industrial platform available to the 30 million Americans living with…

The federal government has awarded Children’s Hospital of Philadelphia $39 million to develop a scalable platform for treating rare genetic, liver-related diseases in infants and children