Lately, CRISPR- mediated genome engineering has been making headlines in newspapers around the world, claiming that this is a revolutionary development that allows scientists to engineer any part of the human genome with extremely accurate precision. CRISPR has been referred to as a milestone in medical science by The Wall Street Journal, The New York Times, Scientist Magazine and more because researchers claim that the technology has the capability to treat a range of diseases from cancer and AIDS, to inherited genetic disorders such as sickle-cell anaemia and Down syndrome. Whether or not CRISPR can be referred to as a “miracle” technology is still up for debate, however, there is no doubt that CRISPR is something worth talking about.
Dr. Rachel Haurwitz began researching CRISPR, or what was then a largely uncharacterized prokaryotic immune system, as a graduate student at UC Berkeley. She recognized the broad commercial uses of several of the proteins from the CRISPR system and in response, co-founded Caribou Biosciences.
“Caribou Biosciences is really focused on broadly developing tools and technologies for cellular engineering and analysis. The corner-stone technology that we are working on now is the Cas9 genome engineering technology.”
Caribou Biosciences, a Berkeley based company, specializes in utilizing the Cas9 enzyme, an extremely efficient genome editing platform.
Dr. Haurwitz, along with co-workers who have extensive biotechnology experience, is working to build a strong R&D team at Caribou to realize the full potential of Cas9-mediated genome engineering by improving specificity, refining efficiency and considering delivery concerns in certain cells and organisms. Caribou Biosciences is already working with commercial partners like SAGE Labs, a mouse and rat model company.
“We have partnered with SAGE to make our technology available to them for the generation of new mouse and rat models and at the same time we are benefiting tremendously from their experience with site-specific genome engineering technologies in the commercial marketplace.”
Caribou Biosciences has the research tools and technologies to provide transformative capabilities to basic and applied research and development in the drug development, agricultural biotechnology, and industrial biosciences fields.
The future of this technology is bright. There is ample opportunity to make custom cell line models for particular diseases. Dr. Haurwitz believes that the industry will quickly realize that there are other significant market opportunities such as engineering microbes for industrial production of molecules, for modifying plants and crops for improved traits and even for the use of human gene therapy to specifically modify cell types to treat patients.
But challenges arise in the field of synthetic biology.
“A big challenge for the synthetic biology industry is actually the term synthetic biology. We as scientists have done a poor job of communicating what that means to the general public and how these technologies are actually critical to improving market areas. There is an unfortunate misperception in the general populous of “frankenfoods” and dangerous technologies and the onus is really on us as a community to overcome that. That is based in education, in doing a better job at explaining what it is that we are doing and building.”
Is there too much hype around CRISPR ?
“I would say that Cas9 , like other site-specific genome engineering technologies such as TALENs( transcription activator-like effector nucleases) or ZFNs ( zinc finer nucleases), does have the capability of being deployed appropriately in a therapeutic context to modify patients cells at the genomic level in order to either repair disease causing genes or otherwise prevent infections. For example Sangamo Biosciences, which is a company here in the Bay Area, has clinical trials ongoing where they are using ZFN technology to modify T-cells or stem-cells in order to knock out the receptor that is necessary for HIV infection. So they are using a site specific genome engineering technology with the ultimate goal of curing HIV. In terms of Cas9 specific therapeutic applications, I think there is tremendous opportunity. I also think realistically it is a long time away from achieving some of what you mentioned, simply thinking about the preclinical and clinical path and timeline that is associated with the development of such therapeutics. I also think the trickiest part is not actually editing the cells. There are a number of technologies for editing the cells; it’s appropriately and safely delivering the editing technology that is the tricky part.
I certainly think it’s incredibly exciting how quickly this technology has been adopted by a number of folks in the research space and we are certainly thrilled by the explosion of it and are excited to be a part of that.”
Dr. Haurwitz is a co-founder of Caribou Biosciences and has been President and CEO since its inception. She has extensive research experience in the field of CRISPRs and has contributed both to the discovery of components of the CRISPR immune system and to the invention of new technologies based on CRISPR-associated proteins.
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