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

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.

The CRISPR-Cas gene-editing system has long been the focus of research as a promising tool in genome editing. However, the ...

The draft guidance supports the agency’s new pathway designed to speed up the development of custom gene therapies.

Scientists have taken an important step toward a gene therapy that could one day turn off the extra genetic material that ...

The document outlines the non-clinical sequencing and bioinformatics data sponsors need to submit as part of INDs and BLAs for gene-editing therapies.

Genomics gains momentum as breakthroughs, falling sequencing costs, and CRISPR advances drive precision medicine and fuel rapid market growth.

A new ACC scientific statement details the potential of gene editing for monogenic cardiovascular conditions like transthyretin amyloidosis.

Scientists have gained tremendous control over the genome with CRISPR editing systems, that use a guide RNA molecule to ...

The rapid evolution of CRISPR/Cas genome editing has redefined the possibilities of cellular and gene therapy, enabling ...