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

In this post, we introduce our CMN disease overview, which will provide information on all the diseases that are targeted by gene-editing therapies...

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...

In 2021, we've seen positive clinical data for the first ever in vivo CRISPR medicines, base editors are heading for the clinic, and CRISPR therapies...

New research on X-linked Agammaglobulinemia has led to the development of a potential gene therapy cure for this disease - or as Dr David Gray...

Professor Waseem Qasim, of University College London and Great Ormond Street Institute of Child Health, helps run early-phase clinical trials using...

Our clinical trials database is a comprehensive collection of the current CRISPR-based trials for gene-editing therapies and diagnostics. Here, we...