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 ...

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

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

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 ...

The deletion of two cancer genes, CHD1 and MAP3K7, improves how well tumors respond to cancer immunotherapy and could be used ...

Current gene therapies to treat sickle cell disease are complex, time-consuming, and are sometimes linked to serious side effects like infertility or blood cancer. To address these challenges, Johns ...

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

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