CRISPR-Cas12a

CRISPR-Cas12a (formerly known as Cpf1) is a type V CRISPR-Cas system utilized for precise genome editing. It is a DNA-guided endonuclease that differs from the more common Cas9 by requiring only a single CRISPR RNA (crRNA) for targeting, recognizing a T-rich protospacer adjacent motif (PAM), and generating staggered double-strand breaks with "sticky ends." These features make it particularly useful for multiplexed gene editing and specific applications in human cells where Cas9 may be limited. In therapeutic development, Cas12a is being explored for gene therapies targeting hematological disorders, such as sickle cell disease and beta-thalassemia, by modulating fetal hemoglobin regulators like BCL11A. It is also a core component of diagnostic platforms due to its collateral cleavage activity.

Other names

Cas12aCas-12aCas 12aCpf1Cpf-1Cpf 1Cas12a endonucleaseCas-12a endonucleaseCas 12a endonuclease