CRISPR/Cas9: short history, big future

Opinion By Nik Sanyal, Ph.D.

If you had to fit the progress in the CRISPR/Cas9 field into a regression model - “exponential growth” would probably be your first choice. In a short span of 14 years, the CRISPR/Cas9 system went from a discovery to integrating paradigm shifting and groundbreaking applications that can change human health, agriculture, ecosystems, and much more. Clustered Regularly Interspaced Short Palindromic Repeats, or CRISPR was originally discovered as DNA sequence that confers adaptive immunity in bacteria against subsequent phage infections. Thereafter the discovery of Cas9, a nuclease that recognizes and cleaves specific CRISPR sequence heralded a new frontier of gene editing and the final pieces of the puzzle were the discovery of CRISPR targeting RNA (crRNA) and trans-activating CRISPR RNA (tracrRNA). This ultimately led researchers to be able to design sequences to match biological targets of interest. By 2017, the CRISPR/Cas9 genome editing system has given us the power of repairing disease mutations in human embryos.

Like the gold rush from the 18th century, the prospects of CRISPR/Cas9 have drawn attention from companies and institutions alike, all wanting to get a big slice of the pie. As soon as the commercial potential of engineered CRISPR/Cas9 emerged, the scramble for licensing intellectual property (IP) began. The University of California- Berkeley, Broad Institute, and researcher Emmanuelle Charpentier hold key IP, who have all licensed out the technology to companies for overlapping applications in human therapeutics, industry, and agriculture. Some of the earliest movers in licensing have been - Caribou Biosciences, Intelllia Therapeutics, ERS Genomics, Editas Medicine, and CRISPR Therapeutics- many of whom further out-license the tailored CRISPR/Cas9 systems for specific applications. The applications already include livestock & agriculture modification, in vitro research tools, and human diagnostics & therapeutics- muscular dystrophy, β-thalassemia, cystic fibrosis, hypertrophic cardiomyopathy, sickle-cell anemia- just to name a few. In theory, any insertion/deletion/mutation is fair game for a CRISPR/Cas9 gene editing system, offering limitless possibilities.

Graph data generated using PubMed search:

Another interesting angle for looking at the progress of CRISPR/Cas9 is its impact and value on society. CrisprCon, a conference that discusses hopes and fears for the technology in a more societal context, took place in University of California, Berkeley during August 2017. Executives of Agriculture stalwarts like Cargill squared off with organic farmers, environmental activists, and the general public to debate about wide effects of introduced genetically modified organisms (GMOs). Till date, most GMO modifications have been done on crops, and driven by convenience to farmers and profits to corporations – think increased shelf life, pest-resistance, etc. – not necessarily an improvement in taste, aroma, and nutritional improvements. CRISPR now provides hopes of more ‘valuable’ crops such as high-nutrition tomatoes, or grapes that make high- resveratrol wine with fewer hangover-causing toxins. Although these discussions of hopes and fear broadly fall under the scope of GMOs, the bright prospects of CRISPR have helped draw a spotlight on long-term effects.

Human health is perhaps the most relevant area to receive long-term benefits from CRISPR/Cas9 modifications. CrisprCon invited discussions between patient-centric entities such as Minority Coalition for Precision Medicine, which is trying to prevent exploitation of racial difference in medical innovation, and Caribou Biosciences, which is at the forefront of precision medicine. Caribou’s co-founder and CRISPR discoverer Jennifer Doudna has embarked on bringing global consensus and active discussions on appropriate clinical uses for CRISPR. As Doudna suggests “I've never seen science move at the pace it’s moving right now…which means we can’t put off these conversations”, reminding us to keep a watch on long-term consequences and responsible use of the CRISPR technology.

Check out some relevant URLs (Click on Article #):

Article 1: Science article – the birth of CRISPR Inc.

Article 2: Cell- Heroes of CRISPR.

Article 3: Wired magazine (CrisprCon)


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