The Alliance for Regenerative Medicine’s (ARM) Gene Editing Task Force on Tuesday released a set of principles for human genome editing endorsed by thirteen of its members who are involved in the ...

The US Food and Drug Administration (FDA) has issued two draft guidances addressing the development of human gene therapy products that incorporate genome editing of human somatic cells and chimeric ...

Scientists have corrected an extremely rare and life-threatening genetic disease of the liver in mouse models and human ...

For decades, the standard explanation for autoimmune diseases like rheumatoid arthritis has rested on two pillars: inherited ...

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.

Cemacabtagene ansegedleucel, an allogeneic chimeric antigen receptor (CAR) T-cell therapy, is being investigated in relapsed/refractory large B-cell lymphoma. The investigational allogeneic chimeric ...

There are an estimated 6,000 Mendelian diseases, most of which still lack cures. Gene editing is an essential technology for studies aimed at generating data about disease-causing gene mutations and ...

9 min read |New research in a HD mouse model points to a key culprit: a small fragment called HTT1a. Lowering HTT1a levels successfully delayed disease signs in mice that model HD, perhaps shaping the ...

A single infusion of a CRISPR-based gene-editing therapy was associated with reductions in LDL cholesterol and triglycerides ...