Base editing offers far more versatility than its name implies. The technique was initially developed to correct ...

Delivering a CRISPR-Cas-derived adenine base editor as a short-lived protein-guide RNA complex enabled efficient PCSK9 ...

The US FDA has accepted Intellia Therapeutics’ application for lonvoguran ziclumeran (lonvo-z, formerly NTLA-2002) and ...

A recent British study at the University of Bristol indicates that the last universal common ancestor (LUCA) possessed an early form of the CRISPR-Cas immune system, dating back approximately 4.2 ...

The company’s lead candidate, AXL-003, is advancing towards preclinical evaluation in an autoimmune indication with liver-targeted delivery. This programme builds directly on results showing that AXL ...

Conventional CRISPR-CAS9 tools trigger immune responses in host animals, which can systematically distort screening results and mask genuine cancer vulnerabilities. A novel platform called StealTHY ...

The team behind Ariya Bio, from left to right: Mierelle Schuijt (Research Associate), Aniruddhan Balakrishnan (Corporate Strategy Intern), Mandy Boontanrart (Cofounder), Maike Getwan (Research Intern) ...

Precision BioSciences reported progress with its CRISPR-Cas–based ARCUS gene editing therapy PBGENE-DMD, designed to excise dystrophin exons 45–55 and restore near full-length dystrophin expression.

Packing chemical modifications throughout the prime editing guide RNA, rather than only at its ends, raised editing in mouse liver to 67% after a... Delivery: Protein-delivered base editor preserves..

A novel engineered TadA ortholog-derived cytosine base editor (aTdCBE) can overcome existing limitations in motif preferences and adenosine activity seen in current cytosine base editors (CBEs). The ...

Intellia Therapeutics has reported positive three-year follow-data from the ongoing Phase 1/2 trial of lonvo-z (formerly known as NTLA-2002) in patients with hereditary angioedema. The data show that ...

Dr. Cecilia Jimenez-Mallebrera, a leading neuromuscular disease researcher, examines how gene editing is progressing from the laboratory to clinical reality for muscular dystrophies. Through selected ...