A new nonprofit wants to streamline gene therapy for diseases often avoided by pharmaceutical companies — making treatment more like a routine procedure than a bespoke drug.
The two-year treatment targeting protein production by disease-causing mutations dramatically reduced seizures and sparked ...
Genetic prediction models are poised to revolutionize medicine. But because they have been trained on European DNA, they ...
Many neurological diseases damage specific parts of the brain, even though the harmful protein involved is present throughout the brain.
Scientists have successfully edited the genome of biting midges, opening the door to new research on how these insects transmit disease. Culicoides biting midges are small blood-feeding insects ...
BACKGROUND: Metabolic adaptation and maladaptation are hallmarks of the failing heart and may be a target for therapeutic ...
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