Spinal muscular atrophy is a severe neuromuscular disease caused by mutations in the SMN1 gene. While a few therapies have been approved for SMA, none...

At a length of around 100 nucleotides, single guide RNAs are not synthesised efficiently. However, thanks to click chemistry, they can now be...

This up-to-date roundup presents the 7 clinical-stage gene-editing approaches to sickle cell disease, which affects millions of people worldwide and...

A CRISPR-based therapy for the metabolic liver disease phenylketonuria (PKU) has moved one step forward. In vivo prime editing in neonate mice...

Researchers at Ulsan National Institute of Science and Technology in South Korea have developed a CRISPR-Cas9-based method to eliminate tumour cells....

This week's update includes news about two gene-edited therapies for cancer. Allogene Therapeutics' ALLO-316 has been granted Fast Track Designation...

Intellia Therapeutics recently announced that the first patient had been dosed in its Phase 1/2a CRISPR trial for acute myleoid leukaemia (AML). The...

For the first time, a team of scientists and medical doctors in Germany is exploring CRISPR to treat the rare genetic disorder Metachromatic...