A new approach for delivering miniature research tools into the interior of egg cells and embryos has been developed, resolving a major bottleneck to using the gene-editing tool CRISPR-Cas9 in many ...
Microinjection will still be the method of choice for delivering CRISPR-Cas9 in many organisms. Penetrance (the percent of cells that successfully take up the CRISPR cargo) can be as high as 90 ...
Transthyretin amyloidosis with cardiomyopathy (ATTR-CM) is a progressive, often fatal disease. Nexiguran ziclumeran (nex-z) is an investigational therapy based on CRISPR-Cas9 (clustered regularly ...
An exon-skipping technique using dual single-guide RNA/Cas9 ribonucleoproteins targeted at 3 novel COL7A1 exons with pathogenic heterozygous mutations achieved exon deletion rates of up to 95%.
Clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated (Cas) proteins are core components of fast-evolving therapeutic gene editing tools. Scientists have used CRISPR ...
Crispr Therapeutics, a leader in CRISPR/Cas9 biotechnology, recently received FDA and EMA approval for Casgevy, the first CRISPR/Cas9-based treatment for TDT and SCD. The partnership with Vertex ...
Announcing a new article publication for Zoonoses journal. As global warming increases, mosquito activity areas are expanding, alongside changes in the natural environment and the misuse of ...
The University of Bayreuth's Biomaterials research group has, for the first time, successfully applied the CRISPR-Cas9 gene-editing tool to spiders. Following the genetic modification, the spiders ...
The acorn worm (Saccoglossus kowalevskii) is an important research organism in evolutionary developmental biology and is collected in Woods Hole. Its eggs are very fragile and they typically don’t ...