Research links variations in the gene GRIN2A to a higher risk of developing schizophrenia and other forms of mental illness. The GRIN2A gene regulates communication between neurons by producing the ...
Scientists have discovered that avian influenza viruses have a gene that makes them incredibly resistant to heat, rendering our body's natural defense system – fever – powerless in fighting infection.
Encoded Therapeutics to Present Interim Phase 1/2 Clinical Data on ETX101, the First One-time Gene Therapy in Development for Dravet Syndrome, at the 2025 American Epilepsy Society Annual Meeting – ...
Credit: Novartis. The approval was supported by data from phase 3 STEER study, which enrolled patients with SMA who were treatment-naive and able to sit but never able to walk independently. Itvisma ...
What Is Itvisma, and Why Does It Matter? Itvisma (onasemnogene abeparvovec-brve) is a gene therapy that the FDA has approved to treat spinal muscular atrophy (SMA) in adults, teens, and children 2 or ...
Researchers have found a new treatment for Huntington's disease, an inherited genetic brain disease that affects over 41,000 Americans, in breakthrough clinical trial that involved at least one ...
A team of researchers at the Broad Institute, led by gene-editing pioneer David Liu, has developed a new genome-editing strategy that could potentially lead to a one-time treatment for multiple ...
The FDA has officially limited the label of Sarepta Therapeutics’ Elevidys, putting an end to a whirlwind few months that saw the abrupt departure—and reinstatement—of top agency official Vinay Prasad ...
Altering a single gene may help people lower dangerously high levels of cholesterol and other fats in the blood, according to new research presented Saturday at the annual meeting of the American ...
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