In this brief roundup, we look at what's going on in the CRISPR Medicine field for Leber congenital amaurosis, an inherited group of rare retinal...
The mechanism our immune system uses to fend off viruses also attacks AAV vectors. Adding telomeric sequences to these vectors helps them to fly under...
The CRISPR gene-editing therapy more than doubled the lifespan in a mouse model by tackling the root cause of Hutchinson-Gilford progeria. The new...
CRISPR is believed to hold the cure for many rare single-gene disorders that are otherwise incurable. Alessia Cavazza’s group at University College...
CRISPR genome editing marches forward as a curative therapy for genetic disorders. Here lead scientist Ayal Hendel talks about how he is developing a...
Using 'dead' dCas9 fused to transcriptional activators researchers wake silent genes to alleviate blindness in a mouse model of retinitis pigmentosa -...
The first clinical trial results indicate that CRISPR is a safe and beneficial gene editing tool in humans. However, full therapeutic translation of...
By combining peptide display and different AAV serotypes, researchers have learned how to make and identify viral vectors that can target and...
