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 …

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 …

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 …

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 …

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 …

The viral delivery of base editors has been complicated by their size and by the limited
packaging capacity of adeno-associated viruses (AAVs). Typically, dual-AAV approaches …

Spinal muscular atrophy (SMA), the leading genetic cause of infant mortality, arises from
survival motor neuron (SMN) protein insufficiency resulting from SMN1 loss. Approved …

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 …

CRISPR-based drugs can theoretically manipulate any genetic target. In practice, however,
these drugs must enter the desired cell without eliciting an unwanted immune response, so …