Genetics

CRISPR treats genetic disorder in adult mammal

Researchers have used CRISPR to treat an adult mouse model of Duchenne muscular dystrophy. This marks the first time that CRISPR has successfully treated a genetic disease inside a fully developed living mammal with a strategy ...

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 ...

Medical research

Using artificial RNA editing to restore genetic code

Various genetic diseases caused by point mutations have no established therapeutic approaches. Prof. Tsukahara and colleagues (Japan Advanced Institute of Science and Technology) are studying a therapeutic method using artificial ...

Genetics

First gene-edited babies claimed in China

A Chinese researcher claims that he helped make the world's first genetically edited babies—twin girls born this month whose DNA he said he altered with a powerful new tool capable of rewriting the very blueprint of life.

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