Two of Opus's seven gene therapy assets are in clinical trials, with additional programs expected to enter the clinic.
A Northwestern Medicine study has uncovered a promising new therapeutic approach for a rare genetic brain disorder, according ...
Aurora Therapeutics, cofounded by Nobel Prize–winning scientist Jennifer Doudna, plans to use gene editing and a new FDA ...
In patients with retinitis pigmentosa (RP), crucial cells in the retina known as rods and cones die over time; night ...
A new CRISPR approach can control genes without cutting DNA, opening a safer path for treating genetic diseases. A newly ...
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RNA gene mutations identified as cause of inherited blindness
Researchers from Radboud university medical center and University of Basel have discovered new genetic causes of inherited ...
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Scientists report new immune insights and targets into LRRK2 mutations in Parkinson's disease
Parkinson's disease (PD) is a debilitating and progressive neurodegenerative disorder caused by the loss of ...
An HIV-derived nucleoside therapy now treats rare genetic diseases by restoring mitochondrial DNA and improving muscle ...
Santhosh Girirajan receives funding from the National Institutes of Health. Genetic inheritance may sound straightforward: One gene causes one trait or a specific illness. When doctors use genetics, ...
Tiffany Fransen, diagnosed with Friedreich's ataxia, faces challenges from this rare disease. The genetic disorder affects ...
Genetic inheritance may sound straightforward: One gene causes one trait or a specific illness. When doctors use genetics, it’s usually to try to identify a disease-causing gene to help guide ...
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