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 ...

Gene therapy treatments for rare diseases are being developed, but getting them out of the lab has proved challenging.

Forbes contributors publish independent expert analyses and insights. Juergen Eckhardt leads Bayer’s impact investment unit, Leaps by Bayer. Some of the year's notable breakthroughs include the first ...

Live Science's health channel editor makes predictions about the medical breakthroughs and public health shifts to come in ...

Tiffany Fransen, diagnosed with Friedreich's ataxia, faces challenges from this rare disease. The genetic disorder affects ...

Scientists at Pacific Northwest Research Institute (PNRI) have overturned a long-held belief in genetics: that inheriting two ...