In his annual letter, Flagship Pioneering’s Noubar Afeyan lays out a choice between near-term “human-made miracles” and a ...

A large genetic screen has revealed how stem cells transform into brain cells, exposing hundreds of genes that make this ...

Gene-editing technologies show great promise for medical treatments and research, with the potential to cure thousands of ...

The startup aims to create a platform for treating extremely rare genetic mutations, armed with CRISPR advances and new regulatory pathways.

The gene therapy success with Baby KJ, along with a new FDA program, have companies more interested in treating rare disease.

Using its economic leverage, California has been forging its own path on scientific research for 25 years. Here’s how.

Victoria Gray spent 34 years battling the debilitating pain of sickle cell disease. Then she volunteered to be the world's first "prototype" for a CRISPR therapy, based on technology invented at UC ...

Funding supports safety studies, manufacturing and clinical planning needed before applying to test the treatment in patients ...

Researchers say discoveries could expand the CRISPR toolbox and lead to more efficient, rapid diagnostic tools for detecting COVID-19, influenza, and RSV.

The AHA recommended whole exome or whole genome sequencing as first-line diagnostic tools for mitochondrial diseases.

In the journal ‘Nature,’ Utah State University biochemists and colleagues report newly discovered details about Cas12a3 ...

A new CRISPR breakthrough shows scientists can turn genes back on without cutting DNA, by removing chemical tags that act ...