Researchers have demonstrated the efficacy of a compact, specific and AAV-compatible CRISPR-Cas13 variant, Cas13bt3, for RNA-targeted gene therapy...

Delivering mRNA via acid-degradable nanoparticles offers a new frontier in prenatal gene editing, setting the stage for treatments targeting the root...

In a move to potentially treat blindness, researchers have used CRISPR-Cas9 technology to permanently disrupt VEGFA expression in the eyes of rhesus...

Researchers have developed a compact, all-in-one CRISPR-dCas9-based repressor platform delivered via AAV, effectively silencing gene expression...

Two research groups are independently pioneering a groundbreaking gene-editing treatment for severe immunodeficiencies, potentially transforming the...

Researchers in the US, Japan and the UK report in Nature Biotechnology that genome editing with Cas9 and adeno-associated virus repair templates leads...

HuidaGene Therapeutics recently announced that the FDA has granted Orphan Drug Designation to its CRISPR-based therapeutic candidate, HG302. The...

Researchers report the treatment of a genetic liver disease in mice through transient prime editor expression.