A major medical milestone took place in May 2025, when doctors at the Children’s Hospital of Philadelphia used CRISPR-based ...
In a 15-patient, Phase 1, first-in-human trial, a one-time CRISPR-Cas9 gene-editing therapy safely reduced LDL cholesterol ...
The approach is “very exciting,” but one expert says a lot more research is needed, especially around safety, before clinical ...
A poorly characterized protein, historically thought to be a chaperon or enzyme, may actually be a key player in prostate ...
Scientists introduced CRISPR to the world as a gene-editing tool in summer 2012, when landmark papers from two independent groups demonstrated how the system could be wielded to make cuts in DNA. Now, ...
CHICAGO – A CRISPR-Cas9-based investigational therapy was linked with a drop in serum transthyretin (TTR) levels in patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM), according to ...
Gene therapy has been on a roller-coaster ride since the first patient was treated 33 years ago. This slow-motion story took another turn this week after a panel of experts potentially set the stage ...
By reactivating a long-lost gene, researchers were able to lower uric acid levels and stop damaging fat accumulation in human ...
WEST LAFAYETTE, Ind. — More than a decade ago, scientists harnessed a bacterial molecular machine that identifies and cuts specific sections of DNA, revolutionizing the ability to edit genes and ...