The expansion of the CRISPR-Cas toolbox is highly needed to accelerate the development
of therapies for genetic diseases. Here, through the interrogation of a massively expanded …

Although RNA-guided genome editing via the CRISPR-Cas9 system is now widely used in
biomedical research, genome-wide target specificities of Cas9 nucleases remain …

Abstract Streptococcus pyogenes (Spy) Cas9 has potential as a component of gene
therapeutics for incurable diseases. One of its limitations is its large size, which impedes its …

The naturally occurring prokaryotic CRISPR-Cas systems provide valuable resources for the
development of new genome-editing tools. However, the majority of prokaryotic Cas …

Compact CRISPR/Cas9 systems that can be packaged into an adeno-associated virus
(AAV) hold great promise for gene therapy. Unfortunately, currently available small Cas9 …

Bacterial CRISPR–Cas systems comprise diverse effector endonucleases with different
targeting ranges, specificities and enzymatic properties, but many of them are inactive in …

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 …

The RNA-guided endonuclease Cas9 is a versatile genome-editing tool with a broad range
of applications from therapeutics to functional annotation of genes. Cas9 creates double …

CRISPR/Cas9 nucleases are widely used for genome editing but can induce unwanted off-
target mutations. High-fidelity Cas9 variants have been identified; however, they often have …

Abstract Background The CRISPR-Cas12a (formerly Cpf1) system is a versatile gene-
editing tool with properties distinct from the broadly used Cas9 system. Features such as …