At a length of around 100 nucleotides, single guide RNAs are not synthesised efficiently. However, thanks to click chemistry, they can now be...
Extended survival times and inhibited growth of patient-derived glioblastoma brain tumours have been achieved in mice with CRISPR-Cas9 mediated...
A CRISPR-based therapy for the metabolic liver disease phenylketonuria (PKU) has moved one step forward. In vivo prime editing in neonate mice...
Most pharmaceuticals don't reach the brain, leaving cognitive impairments due to diseases like mucopolysaccharidoses (MPS) hard to treat. Now, a...
Researchers at Ulsan National Institute of Science and Technology in South Korea have developed a CRISPR-Cas9-based method to eliminate tumour cells....
Folding DNA into compact structures allows for efficient integration of large genes, and this might lead to new therapies for genetic diseases with...
In this week's update, we provide news about Intellia Therapeutics' and Regeneron's ongoing Phase 1 trial for NTLA-2001 in transthyretin amyloidosis....
In this week's clinical trial update, we summarise the ongoing in vivo CRISPR clinical trials for three distinct rare genetic diseases: hereditary...
