Genetics

Gene therapy for the treatment of Huntington's disease

Gene therapy targeting the messenger RNA (mRNA) of the mutated huntingtin gene (HTT) can provide long-lasting therapeutic benefit in Huntington's disease after a single administration. An adeno-associated virus (AAV) gene ...

Medical research

'Unlocking' the potential of viruses to fight cancer

Researchers from the Laboratory of Oncolytic-Virus-Immuno-Therapeutics (LOVIT) at the LIH Department of Oncology (DONC) are working on the development of novel anticancer strategies based on oncolytic viruses, 'good' viruses ...

Medical research

Investigators discover new mechanism to boost RNA therapies

Investigators from the Smidt Heart Institute at Cedars-Sinai have identified how biological pacemaker cells—cells that control your heartbeat—can "fight back" against therapies to biologically correct abnormal heartbeat ...

Genetics

Predicting how splicing errors impact disease risk

No one knows how many times in a day, or even an hour, the trillions of cells in our body need to make proteins. But we do know that it's going on all the time, on a massive scale. We also know that every time this happens, ...

Medical research

Allergic asthma transforms cells of the airway

New research at National Jewish Health shows that allergic asthma fundamentally transforms the human airway, reducing cells' ability to remove pollutants and fight infections. Allergic asthma, the most common form of the ...

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