In May 2025, the world celebrated the success of KJ, an infant who was treated with the world’s first personalized CRISPR gene editing therapy. KJ was born with severe carbamoyl phosphate synthetase 1 ...

If CRISPR stays active too long, it could cut unintended parts of the genome. To reduce this risk, the researchers designed a self-inactivating CRISPR system. This means that CRISPR edits the gene and ...

CRISPR-Cas9 genome editing enzyme. [Artur Plawgo / iStock / Getty Images Plus] We need a patient-first approach for any variant in any patient, whomever, wherever they are. Each and every patient ...

Two papers published in The New England Journal of Medicine highlight the potential of gene editing for treatment of sickle ...

It has been described as a revolutionary technology — and won its inventors a Nobel Prize. CRISPR gene editing, often simply dubbed CRISPR, is a tool that allows scientists to precisely target and ...

LOS ANGELES (KABC) -- It's been more than a month since the FDA approved two milestone gene-editing treatments for sickle cell disease, but lining up patients for these therapies will be a challenge ...

The gene editing treatment targets the relatively common 1717-1G>A splicing mutation, which does not respond to many current ...

Researchers announced two major CRISPR breakthroughs: a method to boost production of virus-like particles for gene delivery, and a ThermoCas9 enzyme that can distinguish cancer cells from healthy ...

Researchers have developed a modified CRISPR gene-editing tool with potential to silence the extra chromosome causing Down ...