A compact Cas12f nuclease shows high editing efficiency in human cells, with structural insights enabling an engineered variant potentially suited for future AAV‑compatible delivery.

For the millions of people living with genetic diseases like muscular dystrophy and inherited liver disorders, one of the ...

A research team has discovered an enhanced CRISPR gene-editing system that could enable targeted delivery inside the human ...

The US Food and Drug Administration (FDA) today issued a draft guidance for sponsors seeking approval of human gene therapy ...

A study showed that base editing can repair mutations linked to a rare liver disease. The therapy restored liver and cellular ...

NIH-funded UT team and colleagues from Metagenomi Thereapeutics discovered highly efficient enzyme could enable targeted gene editing within the human body.

A number of the world’s top scientists, policy experts and bio-ethicists met this week in Washington, DC to discuss the implications of human gene editing, and though the focus of the summit was ...

Researchers used CRISPR to edit hematopoietic stem cells, creating B cells that produce neutralizing antibodies against HIV, ...

The US Food and Drug Administration (FDA) has released draft guidance on how sponsors can utilize next-generation sequencing ...

A multidisciplinary committee of experts studied the scientific underpinnings of human gene-editing technologies, their potential use in biomedical research and medicine -- including human germline ...