Wave Life Sciences has reached alignment with the US Food and Drug Administration on a registrational pathway for WVE-006, a GalNAc-conjugated RNA...
A mutation-agnostic CRISPR-Cas strategy that inserts a functional rhodopsin coding sequence into the human RHO locus has preserved retinal structure,...
A nuclease-inactive CRISPR-Cas9 system increased endogenous expression of two protective HDL components without cutting genomic DNA. In a small mouse...
Delivering a CRISPR-Cas-derived adenine base editor as a short-lived protein-guide RNA complex enabled efficient PCSK9 editing while human embryos...
Using microneedles for transdermal delivery of CRIPSR-Cas9 and glucocorticoids researchers have found a viable way to precisely treat inflammation...
Pharmaceutical and biological research as well as biologics manufacturing rely on genetically modified cell lines with genes that have been modified...