Why Crispr-Cas9 is Pandora’s Box
A decade ago, zinc-fingers nuclease (ZFN) was the tool used for genomic editing. It was expensive, difficult to engineer, and therefore not widely utilized. Then came TALENs, transcription activator-like effector nucleases, enzymes that could be engineered to edit DNA. Both methods were costly, complex to execute and had limiting factors.
In 2012, a major breakthrough in genomic editing was announced. Jennifer Doudna at the University of California, Berkeley, and microbiologist Emmanuelle Charpentier of the Max Planck Institute for Infection Biology in Berlin and Umeå University in Sweden, published a research paper [1] that showed that the Cas9 enzyme can be directed to cut specific sites in isolated DNA. In the following year, Feng Zhang at the Broad Institute of MIT and Harvard in Cambridge, Massachusetts, published research demonstrating the use of Crispr-Cas9 for genetic editing in cells of mammals [2].
Crispr-Cas9 is the breakthrough technology that enables researchers to rearrange genes quickly and easily. Crisper stands for Clustered regularly-interspaced short palindromic repeats, a description of the genetic basis of the technique. Cas9 is the name of a protein that enables inexpensive genetic editing.
Teams of researchers around the world are currently using Crispr-Cas9 to experiment on a wide variety of projects. For example, researchers are testing ways to treat and cure diseases and engineer disease-resistant crops using Crispr-Cas9. But with this powerful new technology comes possible ethical issues such as human germline editing, the altering of the DNA of heritable traits in humans. The possible consequences of editing germline traits are not known.
Like Pandora’s box, with the release of the potential for harm, there is hope for curing a myriad of diseases such as cystic fibrosis, muscular dystrophy, Huntington’s disease, Alzheimer’s, and Parkinson’s disease. With this hope comes economic opportunity.
Many startups based on Crispr-Cas9 have recently emerged. According to The Boston Consulting Group, gene-editing startups have attracted over $1 billion in private venture-capital investments since 2013.
Intellia Therapeutics, formed in 2014 with licensed technology from Caribou Biosciences, has raised $155 million from various investors, including OrbiMed, Atlas Venture and Novartis.
Editas Medicine Inc., a Cambridge biotech startup with investors that include Google Ventures and Bill Gates, raised $163.3 million from sales of preferred stock.
German pharmaceutical group Bayer AG established a joint venture with Crispr Therapeutics AG with a plan to invest a minimum of $300 million over a five year period. Swiss-based Crispr Therapeutics Ltd.’s CEO, Rodger Novak has said he would consider an IPO in 2016 [3].
Genetic research has come a long way from the days of Gregor Mendel, the father of modern genetics from the region what is today known as the Czech Republic. Innovation in molecular biology is bringing us to the precipice of redefining the genetic composition of any living organism. Crispr-Cas9 is disruptive technology that will revolutionize biotech, food, pharmaceutical, healthcare and more industries along its path.
Copyright © 2016 Cami Rosso All rights reserved.
References
- Science, Vol. 337, Issue 6096, 17 Aug 2012, pp. 816–821.
- Science, Vol. 339, Issue 6121, 15 Feb 2013, pp. 813–823.
- Bloomberg Business, January 4, 2016.
Originally published at https://www.linkedin.com on March 22, 2016.
