Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated
protein 9 (Cas9) gene-editing technology is the ideal tool of the future for treating diseases …

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 …

Cytosine base editors (CBEs) are larger and can suffer from higher off-target activity or lower
on-target editing efficiency than current adenine base editors (ABEs). To develop a CBE that …

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 …

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 …

The ever-expanding set of CRISPR technologies and their programmable RNA-guided
nucleases exhibit remarkable flexibility in DNA targeting. However, this flexibility comes with …

Abstract The type VF CRISPR-Cas12f system is a strong candidate for therapeutic
applications due to the compact size of the Cas12f proteins. In this work, we identify six …

Despite the availability of Cas9 variants with varied protospacer-adjacent motif (PAM)
compatibilities, some genomic loci—especially those with pyrimidine-rich PAM sequences …

As the evolutionary ancestor of Cas12 nuclease, the transposon (IS200/IS605)-encoded
TnpB proteins act as compact RNA-guided DNA endonucleases. To explore their …