RNA therapeutics have had a tremendous impact on medicine, recently exemplified by the
rapid development and deployment of mRNA vaccines to combat the COVID-19 pandemic …

According to Darwin's theory, endless evolution leads to a revolution. One such example is
the Clustered Regularly Interspaced Palindromic Repeats (CRISPR)–Cas system, an …

The diversity, modularity, and efficacy of CRISPR-Cas systems are driving a
biotechnological revolution. RNA-guided Cas enzymes have been adopted as tools to …

Duchenne muscular dystrophy (DMD) is a fatal muscle disease caused by the lack of
dystrophin, which maintains muscle membrane integrity. We used an adenine base editor …

Duchenne muscular dystrophy (DMD) is a severe, progressive, and ultimately fatal disease
of skeletal muscle wasting, respiratory insufficiency, and cardiomyopathy. The identification …

Mutations in the gene encoding dystrophin, a protein that maintains muscle integrity and
function, cause Duchenne muscular dystrophy (DMD). The deltaE50-MD dog model of DMD …

The development of clustered regularly interspaced short-palindromic repeat (CRISPR)-
based biotechnologies has revolutionized the life sciences and introduced new therapeutic …

Ischaemic heart disease is a leading cause of death worldwide. Injury to the heart is
followed by loss of the damaged cardiomyocytes, which are replaced with fibrotic scar …

The introduction of insertion-deletions (INDELs) by non-homologous end-joining (NHEJ)
pathway underlies the mechanistic basis of CRISPR-Cas9-directed genome editing …

The introduction of induced pluripotent stem cells (iPSCs) has opened up the potential for
personalized cell therapies and ushered in new opportunities for regenerative medicine …