The New England Journal of Medicine, in its Clinical Implications of Basic Research column, covered a remarkable new study that shows just how revolutionary and pathbreaking the CRISPR-Cas9 gene may be.
The column -- and the research it is based upon -- integrates across the seemingly disparate field of retrovirology and xenotransplantation. Xenotransplantation is the transplantation of organs from an animal species into humans. Even if a safe and effective technique that minimized organ rejection could be found, however, the problem of xenozoonosis would still exist.
Xenozoonosis is the transmission of an infectious agent via organ transplantation from one species to another. While screening donor animals for exogenous potential pathogens and raising them in sterile environments may be possible, it is not enough. Endogenous retroviruses integrated into the genomes of animals could activate and infect human cells causing novel zoonotic diseases. The fact that these viruses are literally part of the organism whose organs are being transplanted is a very daunting obstacle to overcome. But not for CRISPR-Cas9.
CRISPR-Cas9, a revolutionary gene-editing technique that is part of bacteria's immune system, can be used to cut and paste genes in a relatively simple manner. In the study (originally published in Science by Yang along with transplant infectious disease pioneer Jay Fishman and the extremely innovative George Church) detailed in The New England Journal of Medicine, CRISPR-Cas9 was employed successfully to remove porcine endogenous retrovirus (PERV) from pig cell lines. For many reasons, pigs will be the most likely source of organs for humans and PERV is known to have the capacity to infect human cells.
While this is an early study, its implications are far-reaching -- just imagine the impact on organ transplantation waiting list if pig organs could be safely transplanted into humans.
CRISPR-Cas9 may be the discovery of the millenium.