CMV-LbCpf1 AAV (Serotype 9)

CMV-LbCpf1 AAV is an adeno-associated virus (AAV) vector specifically designed to express the LbCpf1 (also known as Lachnospiraceae bacterial Cpf1) nuclease using a cytomegalovirus (CMV) promoter. AAV serotype 9 is widely used in gene therapy and research due to its ability to infect a wide range of cell types and tissues. Serotype 9 vectors are particularly known for their efficient transduction in liver, muscle, and central nervous system cells, which are common targets for therapeutic delivery of gene editing tools in preclinical and clinical studies.

LbCpf1 is part of the CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) system, a powerful gene editing tool. Unlike the more widely known Cas9, Cpf1 has unique properties that make it attractive for a variety of applications. Compared to Cas9, LbCpf1 is able to recognize different protospacer adjacent motif (PAM) sequences, providing researchers with greater flexibility in genome editing. It also produces staggered DNA breaks, which is highly advantageous for certain repair and insertion applications. The CMV promoter is a strong promoter that drives high-level expression in a variety of cell types. Combining the CMV promoter with LbCpf1 in an AAV serotype 9 vector can fully utilize the advantages of both the promoter and the delivery system to achieve efficient in vivo or in vitro experiments.