Market Overview

CRISPR Researchers Target Incurable Blood Cancer


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. Core members of the Innovative Genomics Initiative on the UC Berkeley campus and affiliated laboratories at Bay Area universities and The Broad Institute and its partner institutions at MIT, Harvard and associated hospitals participated in this initiative.

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 (Sloan-Kettering), Ruben Mesa (The Mayo Clinic-Scottsdale), Andrew Schafer (Weill-Cornell), John Crispino (Northwestern) and Richard T. Silver (Weill-Cornell) 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.

MPN Genetics Network
Stephanie Onel, 828-279-3861

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