Genetics

Study encourages cautious approach to CRISPR therapeutics

A comprehensive study—conducted by researchers at Sanford Burnham Prebys, the National Cancer Institute (NCI) and other groups—has shown that gene editing, specifically gene knockout (KO), with CRISPR -Cas9 can favor ...

Ophthalmology

A new protein treatment for glaucoma?

A Northwestern Medicine study in mice has identified new treatment targets for glaucoma, including preventing a severe pediatric form of glaucoma, as well as uncovering a possible new class of therapy for the most common ...

Neuroscience

Scientists take major step toward Angelman Syndrome gene therapy

Babies born with a faulty maternal copy of the UBE3A gene will develop Angelman syndrome, a severe neurodevelopmental disorder with no cure and limited treatments. Now, for the first time, scientists at the UNC School of ...

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