An HIV-derived nucleoside therapy now treats rare genetic diseases by restoring mitochondrial DNA and improving muscle ...

A new CRISPR approach can control genes without cutting DNA, opening a safer path for treating genetic diseases. A newly developed form of CRISPR at UNSW Sydney points to a safer way of treating ...

With a $125 million series B financing round, Diagonal Therapeutics has made a vertical move to advance its first-in-class ...

Many genetic variants linked to inherited eye disorders rarely lead to disease, meaning up to 2% of people could carry these variants without ever developing vision problems, according to a new s tudy ...

A Northwestern Medicine study has uncovered a promising new therapeutic approach for a rare genetic brain disorder, according ...

This color-coded graphic shows different populations of cells in the mouse brain, each one targeted by one of the genetic tools developed by scientists at the Allen Institute and other institutions.

A research team led by Zhiping Weng, Ph.D., and Jill Moore, Ph.D."18, at UMass Chan Medical School, has nearly tripled the ...

University of Otago – Ōtākou Whakaihu Waka has led international research uncovering a new genetic cause for a rare developmental disorder that profoundly impacts brain growth and function in children ...

Khaberni - A massive scientific study published by 'Nature' magazine revealed that many psychological disorders, typically ...

Every time you look in the mirror, you are seeing the legacy of an extinct cousin. A small but influential fraction of your genome comes from Neanderthals, and those ancient fragments are still ...