Science & Medicine

News: CRISPR comes to MPNs.

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  Contact:  Stephanie Onel, 828-279-3861


CRISPR researchers target incurable blood cancer.

Collaborative efforts launch gene editing research into myeloproliferative neoplasms.


Asheville, North Carolina, April 7, 2015 … The thorny task of bringing together two separate and highly technical professional disciplines – hematology/oncology and genomic engineering –has crossed its first major hurdle.

In the first quarter of 2015, in a coordinated effort driven by the MPN Research Foundation, MPN patients set out to raise awareness of their rare and incurable cancer among CRISPR/Cas9 researchers.

MPN patient advocates personally introduced genetic engineering scientists to the challenges and research opportunities in myeloproliferative neoplasms.  Meetings were held with Jennifer Doudna, George Church, Feng Zhang, Emmanuelle Charpentier, Rachel Haurwitz and many other pioneers in the discovery and development of CRISPR/Cas9 gene editing.

One result:  Several CRISPR/Cas9 researchers have now designed MPN research projects ranging from targeting selected mutations and gene conversion to deployment of virus-based delivery systems..

Collaboration in MPN gene editing research has been driven by initiatives in both the genetic engineering and hematology/oncology communities.

The MPN Research Foundation, in Chicago, funded mGEN, the MPN Genetics Network  in December 2014 as a resource to help bridge these worlds.  mGEN project leader is Zhenya Senyak, an MPN patient advocate and editor of MPNforum and the MPN Quarterly Journal. -.

The Foundation helped jumpstart the union of genetic engineering and hematology by adding, with the support of its partner, the Leukemia and Lymphoma Society, a series of $200,000 challenge grants to this year’s award cycle. Those competitive grants attracted proposals from leading CRISPR/Cas9 centers in Europe and the US.

Among scientists and engineers, collaboration has been part of the gene editing environment from the beginning. The Innovative Genomics Initiative on the UC Berkeley campus and affiliated laboratories at Bay Area universities —  actively collaborates with academic and industrial research partners worldwide. IGI founder is CRISPR/Cas9 discoverer Jennifer Duodna.

The primary mission of the Broad Institute and its partner institutions at MIT, Harvard and associated hospitals is to transform medicine through collaborative work in the biological sciences. A core Broad member is Feng Zhang, a CRISPR/Cas9 pioneer.

The Scientific Advisory Board of mGEN played a major supportive and collaborative role in helping shape several MPN CRISPR/Cas9 research proposals. Drs. Ross Levine, Ruben Mesa, Andrew Schafer, John Crispino and Richard T. Silver all directly assisted CRISPR researchers by providing MPN background, support and access to needed materials like tissue samples.

MPNs, initiated by one or more gain of function mutations in hematopoietic precursor cells, are a specially challenging target.  Until now, the goal of therapy has been to relieve symptoms of the disease and inhibit expression of known mutated genes.  Genomic therapy holds promise to eliminate the disease state by editing the underlying mutations.  CRISPR molecules with multiple associated nucleases are ideally suited to knock out several pathogenic mutations in a single operation.

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TAGS:   Blood cancer, gene editing. Medical collaboration. myeloproliferative neoplasm,. MPN Research Foundation,. CRISPR/Cas9,.  mGEN MPN Genetics Network.  Innovative Genomics Initiative. The Broad Institute.  Jennifer Doudna. George Church.Feng Zhang Emmanuelle Charpentier.

Comments on: "News: CRISPR comes to MPNs." (3)

  1. I have polycythemia Vera and is this in clinical trial? I would be interested if so. I was diagnosed 4/5/2006 and this is exciting news.

    • The early application of CRISPR/Cas9 is ex vivo, improving gene therapy (like CAR-T immunotherapy) through more precise selection and repair of genes, etc. Your best — and only official — source for clinical trial data is With PV, you’re likely JAK2+. There are several CRISPR-related JAK2 studies in progress that will report in 2016. Stay active and healthy, our time is coming!

  2. Dave Dickover said:

    Thanks. I look forward to progress. I am fighting AML after 10 years of MF. Keep up all the good works.

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