Messenger RNA (mRNA) is an emerging class of therapeutic agent for the prevention and
treatment of a wide range of diseases. The recent success of the two highly efficacious …

Our ability to edit genomes lags behind our capacity to sequence them, but the growing
understanding of CRISPR biology and its application to genome, epigenome and …

Genome editing has been a transformative force in the life sciences and human medicine,
offering unprecedented opportunities to dissect complex biological processes and treat the …

Realizing the promise of prime editing for the study and treatment of genetic disorders
requires efficient methods for delivering prime editors (PEs) in vivo. Here we describe the …

Biomedical research is undergoing a paradigm shift towards approaches centred on human
disease models owing to the notoriously high failure rates of the current drug development …

In vivo gene editing therapies offer the potential to treat the root causes of many genetic
diseases. Realizing the promise of therapeutic in vivo gene editing requires the ability to …

RNA-based gene therapy requires therapeutic RNA to function inside target cells without
eliciting unwanted immune responses. RNA can be ferried into cells using non-viral drug …

Dominant missense pathogenic variants in cardiac myosin heavy chain cause hypertrophic
cardiomyopathy (HCM), a currently incurable disorder that increases risk for stroke, heart …

Methods to deliver gene editing agents in vivo as ribonucleoproteins could offer safety
advantages over nucleic acid delivery approaches. We report the development and …

RNA therapeutics show a significant breakthrough for the treatment of otherwise incurable
diseases and genetic disorders by regulating disease-related gene expression. The …