The gene editing tools based on CRISPR/Cas9 include CRISPR knockout (CRISPR KO), CRISPR interference (CRISPRi), and CRISPR activation (CRISPRa). The action mechanism of CRISPR/Cas9 in cells in three ...
Microbiologist John van der Oost of Wageningen University & Research (WUR) has received an ERC Proof of Concept grant to further develop a promising CRISPR-based approach to cancer treatment. With ...
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 ...
Dublin, Ireland, and Cheshire, UK, 04 September 2024: ERS Genomics Limited (‘ERS’), the CRISPR licensing Company, and Medicines Discovery Catapult (‘MDC’), an independent, not-for-profit drug ...
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%.
Picture CRISPR-Cas9, a gene editing technology, as a GPS-guided scalpel: gRNA directs the Cas9 enzyme, a protein that cuts DNA, to an exact place in the genome. Before the cut is made, built-in ...
AI-enabled protein design company Profluent has leveraged artificial intelligence to design an open-source gene editor called OpenCRISPR-1, demonstrating the technology can be used to create molecules ...
Most approved gene therapies today, including those involving CRISPR-Cas9, work their magic on cells removed from the body, after which the edited cells are returned to the patient. This technique is ...