Over the past decade, CRISPR has become as much a verb as it is an acronym,
transforming biomedical research and providing entirely new approaches for dissecting all …

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 …

Cell-based therapeutics are an emerging modality with the potential to treat many currently
intractable diseases through uniquely powerful modes of action. Despite notable recent …

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 …

The development of new CRISPR–Cas genome editing tools continues to drive major
advances in the life sciences. Four classes of CRISPR–Cas-derived genome editing agents …

Gene therapy would benefit from a miniature CRISPR system that fits into the small adeno-
associated virus (AAV) genome and has high cleavage activity and specificity in eukaryotic …

Genome editing, which involves the precise manipulation of cellular DNA sequences to alter
cell fates and organism traits, has the potential to both improve our understanding of human …

Gene editing that makes target gene modification in the genome by deletion or addition has
revolutionized the era of biomedicine. Clustered regularly interspaced short palindromic …

The prokaryote-derived CRISPR–Cas genome editing systems have transformed our ability
to manipulate, detect, image and annotate specific DNA and RNA sequences in living cells …

Mutations in RNA binding motif protein 20 (RBM20) are a common cause of familial dilated
cardiomyopathy (DCM). Many RBM20 mutations cluster within an arginine/serine-rich (RS …