Two of the largest science institutions in the country are locking horns over control of a new gene editing technology called CRISP – Cas9. The winner in the legal battle between MIT and University of California Berkeley will emerge with control of one of the most promising gene editing technologies known today.
Background on the CRISPR – Cas9 controversy
Dr. Jennifer Doudna, a biochemist at the University of California, Berkeley, helped make a major discovery back in 2011: a relatively simple method for altering any organism’s DNA. Of note, other current methods are slow, and can result in more off-target effects.
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The discovery has turned Dr. Doudna into a celebrity of sorts, and she has received academic praise and prizes. The CRISPR- Cas9 gene editing technique is already used in lab studies, and the technique may eventually allow the repair of flawed genes in humans, making it possible to treat or even cure a broad range of genetic diseases.
However, the first major patents for the new technology were granted to Feng Zhang, a scientist at the Broad Institute and M.I.T.
In its patent filings, the Broad Institute argues that the paper by Doudna and Charpentier in 2012 did not make it clear how to alter DNA in cells with nuclei (esp. human cells), which required the inventive steps taken by Zhang. His patent application included pages from a lab notebook highlighting that he was researching CRISPR genome editing before the 2012 paper was published.
UC Berkeley is challenging the decision of the Patent Office, and the ongoing legal battle is dampening enthusiasm and stressing collegiality in the field.
Developing the CRISPR – Cas 9 technology
In late 2011, Doudna, French colleague Emmanuelle Charpentier and postdoc researchers Martin Jinek and Krzysztof Chylinski eventually discovered how to join two pieces of RNA using a protein made by a bacteria called Cas9 to cut DNA. Most importantly, the researchers determined that the two RNA pieces could be combined into a single piece and still function.
It was then the scientists made the realization that this cellular defense system could also be used to edit genomes.
Statement from Jennifer Doudna
“I really want to see this technology used to help people,” Doudna said in an interview with the New York Times. “It would be a shame if the I.P. situation would block that.”
“I remember thinking this is probably the most obscure thing I ever worked on,” Doudna said, recalling when she first started on the CRISPR research.
Zinc Finger Nucleases
Another promising gene editing technology is based on zinc finger nucleases. Zinc finger nucleases, or ZFNs, are engineered DNA-binding proteins that enable targeted editing of the genome by creating double-strand breaks in DNA at specified locations. Unlike CRISPRs, ZFNs have already been used and proven safe in human genome editing applications.
ZFN technology offers numerous advantages including immediate disruption of or integration into any genomic loci, mutations that are permanent and heritable, working in a variety of mammalian somatic cell types, making gene edits through a single transfection, achieving knockout or knock-in cell lines in as little as two months, and finally, single or biallelic edits are found in 1–20% of resulting clone population.