CRISPR gene editing technology to be evaluated for its applicability and safety for blood cancer group myeloproliferative neoplasms

February 10, 2015 (Updated October 2015)

One of the primary goals of the MPN Research Foundation is to catalyze new avenues of research into the cause(s) and potential treatments for the blood cancer group polycythemia vera, essential thrombocythemia and myelofibrosis. As an increasing number of new approaches to cancer research are emerging, the Foundation has a new project which hopefully will ensure any applicable technology is tested in MPNs.

One such approach to cancer treatment is CRISPR-cas9, an acronym for Clustered Regularly Interspaced Short Palindromic Repeats. CRISPR-cas9 allows for the targeting of specific mutations. This approach is currently being tested in cystic fibrosis, sickle cell anemia, and some cancers. It is believed to be particularly effective for diseases associated with single point genetic mutations, which suggests that it may be applicable to MPNs, for which there are several known genetic mutations suck as JAK2V617F and CALR.

Currently MPNs are under-represented among the conditions being studied by CRISPR researchers. In addition, not enough is known about the potential impact of this treatment on the people living with MPNs. To ensure that MPNs are sufficiently explored by the CRISPR research world the MPN Research Foundation will be taking action on two fronts:

• Initiate a small investigatory project
• Add CRSISPR as one of our grant focus areas for 2015. We funded 3 projects, each for 2 years:

George Church, PhD Click here for project details
Harvard Medical School
“Establishment of isogenic human induced pluripotent stem cell (hiPSC) lines containing CRISPR engineered MPN mutations”

Zhijian Qian, PhD and Wen-Shu Wu, PhD Click here for project details
University of Illinois at Chicago
“Correction of JAK2 mutation in myeloproliferative neoplasms by gene editing”

Zhaohui Ye, PhD Click here for project details
Johns Hopkins
“Precise Genome Editing for Targeting Malignant Clones in MPNs”

As a cautionary note, this technology, although exciting and currently the subject of much scientific and commercial activity, is in its preliminary stages. The long-term costs and benefits are not yet clear. But given its potential to fundamentally alter the course of MPNs and other diseases, the Foundation believes that further study is critical. The RFP is scheduled to go out in the end of February. To be added to our RFP distribution list send us an email with your contact info.

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