GENE202 is a single dose gene therapy for patients with rare metabolic disease, methylmalonic acidaemia.

A "gene silencer" (technically known as small interfering RNA, or siRNA), locally delivered by nanoparticles embedded in an ...

At a Food and Drug Administration cell and gene therapy (CGT) roundtable last June, most speakers pushed for the agency to ...

Alzheimer’s may be driven far more by genetics than previously thought, with one gene playing an outsized role. Researchers ...

Sometimes, in genetics, two wrongs do make a right. A research team has recently shown that two harmful genetic variants, ...

Thanks to genetic science, gene editing, and techniques like cloning, it’s now possible to move DNA through time, studying ...

New one-dose treatment edits a gene in the liver, disabling the ANGPTL3 protein and significantly lowers cholesterol.

No gene acts alone: interacting variants and protein partnerships can worsen, mask or even rescue disease risk, demanding ...

GEN’s first virtual event of 2026—we present a group of outstanding researchers and thought-leaders to discuss the latest advances and challenges in delivering genetic therapies.

We know the genes, but not their functions—to resolve this long-standing bottleneck in microbial research, a joint research ...

A new study led by researchers at UTHealth Houston investigated both gene expression and regulation at single cell levels to ...

ELRIG, a not-for-profit, volunteer-led organisation dedicated to the global drug discovery community, today announced the ...