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CRISPR, Editas Medicine and Intellia Fall as Scientific Paper Warns of Unintended Mutations Linked to Gene Editing

Written by: | support@biospace.com | Dated: Wednesday, May 31st, 2017

 

May 31, 2017
By Alex Keown, BioSpace.com Breaking News Staff

 


BOSTON – Are there unintended consequences of gene editing? A paper published in Nature Methods indicates there are and that news sent share prices of CRISPR, Editas Medicine (EDIT) and Intellia (NTLAA) tumbling.

The three companies, all using CRISPR-Cas9 gene editing technologies, saw prices fall after the paper’s authors reported unexpected mutations in vivo after CRISPR-Cas9 treatments. The paper’s authors followed up on mice that had been cured of blindness using the gene-editing technique. What they found might give pause to some potential patients. This morning, the Boston Business Journal said the study authors found that two of the mice cured of blindness “experienced a total of more than 1,500 smaller mutations and over 100 larger mutations, none of which had been predicted by computer algorithms.”

Those types of unintended mutations have sparked the interest of Hollywood. Last year, BioSpace reported on a potential television program highlighting some of those concerns about gene-editing. The proposed show, C.R.I.S.P.R., has more of a science fiction premise, but it certainly highlights concerns.

It’s those concerns that Editas and others have sought to allay. In a statement issued to the Boston Business Journal, Editas said developing “safe and effective” treatments using CRISPR techniques is the goal of the company.

“”Part of how we do that is to rigorously assess the specificity of our molecules. We’ve published extensively on our approach to specificity and have demonstrated that we can make CRISPR molecules with no detectable off-target effects,” Editas said in its statement.

Despite the mice mutations, Vinit Mahajan, a professor at Stanford University and one of the co-authors of the Nature Methods paper, told the Journal that researchers are “upbeat” about the potential of gene editing technology.

“We’re physicians, and we know that every new therapy has some potential side effects,” he said, according to the Journal. “But we need to be aware of what they are.“

So far, at least in the United States, CRISPR-Cas9 has not been used in human trials. However, last year researchers in China became the first to inject cells that contained edited genes using CRISPR-Cas9 technology into a human patient. At the end of October an oncology team at Sichuan University in Chengdu dosed a patient with an aggressive form of lung cancer with the edited genes.

In the United States, the Parker Institute for Cancer Immunotherapy has plans to initiate the first CRISPR trial in human patients. The gene editing technology will be used to alter a patient’s blood cells, particularly the T cells, in order to kill the cancer cells.

While CRISPR researchers continue to move toward human testing, there is still an ongoing legal fight over who owns the patents for the technology. In February, the U.S. Patent Office ruled the Broad Institute of Harvard University and the Massachusetts Institute of Technology were the owners. That decision is being appealed by the University of California, the Journal noted.

“CRISPR” refers to Clustered Regularly Interspaced Short Palindromic Repeats that occur in the genome of certain bacteria, from which the system was discovered. Cas9 is a CRISPR-associated endonuclease (an enzyme) known to act as the “molecular scissors” that cut and edit, or correct, disease-associated DNA in a cell. Gene therapy essentially transforms cells inside a patient to harness their immune system to fight an invading disease on its own. CRISPR-Cas9 is considered revolutionary technology, and as such is likely, at some level, to be used by many companies and institutions.

Initial work on CRISPR was done by UC Berkeley professor Jennifer Doudna and Emmanuelle Charpentier. The technology was first described by Doudna and Charpentier in the journal Science in 2012. However, Feng Zhang, a researcher at the MIT-Harvard Broad Institute, was the first to win a patent following his submission of laboratory notes to prove he was the first inventor.

 

 

BioSpace source:

http://www.biospace.com/News/crispr-editas-medicine-and-intellia-fall-as/458185/source=TopBreaking?intcid=homepage-seekernewssection-tabtopbreakingnews

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