CRISPR

CRISPR/Cas

Technology

CRISPR technology, developed from a bacterial enzyme system that recognizes and edits Clustered, Regularly Interspaced Short Palindromic Repeats, and which protects bacteria from viruses, also enables the precise manipulation of genetic information and has the potential to provide substantial benefit. As the technology has matured, CRISPR gene editing has started advancing from ex vivo applications (manipulation of DNA in cells removed from the body which are then reimplanted in the body) to in vivo applications (manipulation of DNA in a patient by infusion of CRISPR enzyme systems into the body).

Origin

Because the system is bacterial in origin, a proportion of treated patients may mount an immune response against the enzyme — pre existing adaptive immunity was very clearly shown in 2019 by Charlesworth et al in Nature Medicine https://www.nature.com/articles/s41591-018-0326-x. At a minimum this response could sharply reduce the utility of this system, requiring pre-screening of prospective recipients or prophylactic immune suppression. Cyrus and The Broad Institute in Cambridge, MA are collaborating on the deimmunization of CRISPR systems with the goal of eliminating the risk of such immune responses.

Origin

Because the system is bacterial in origin, a proportion of treated patients may mount an immune response against the enzyme — pre existing adaptive immunity was very clearly shown in 2019 by Charlesworth et al in Nature Medicine https://www.nature.com/articles/s41591-018-0326-x. At a minimum this response could sharply reduce the utility of this system, requiring pre-screening of prospective recipients or prophylactic immune suppression. Cyrus and The Broad Institute in Cambridge, MA are collaborating on the deimmunization of CRISPR systems with the goal of eliminating the risk of such immune responses.