Science & Medicine

Spring, 2015

MPN milestone: CRISPR gene editing 


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CRISPR labs take on MPN research

Zhen Cropped Team Leader



Most enterprises don’t have such a clear beginning,  but this one clocked in December 8, 2014. 9:27AM, precisely.
It started in a parking lot up above the Greek Theater on the UC Berkeley Campus. And there’s a date stamped parking pass to mark the event.  It’s not every day a crusade has such a clear marker.
In pursuit of an MPNforum story I had come to Berkeley to see Jennifer Doudna, co-discoverer of the CRISPR/Cas9 (Clustered regularly interspaced short palindromic repeats and the CRISPR-associated enzyme) gene editing system.

Across the Bay in San Francisco, the world of hematologists gathered at the annual meeting of the American Society of Hematology.  For decades, the ASH meetings had been  the focus of all our hopes for relief, for a cure.  As I pulled into the  parking lot I was well aware of how symbolic it was to  turn away from those proceedings, even briefly, to explore this unknown territory.

Berkeley Parking PassWith few new drugs to treat our blood cancer — and none dependably effective —  we had been forced to watch friends fall to myelofibrosis and its blast-phase endpoint, acute myeloid leukemia.

Suddenly, there was hope, a fresh approach to treat disease that might apply to MPNs. Now there was a technology coming out of the biology labs that would no longer rely on putting out fires downstream of the blaze.  This technology promised the possibility of correcting mutations that cause the conflagration of myeloproliferative neoplasm at their very source.

Four months later:    CRISPR scientists are now working with MPN cell lines and stem cells as the big guns of structural and synthetic biology have been trained on MPN research projects. 

It took the combined work of patients, hematologists, gene editing scientists, the MPN Research Foundation and MPN Genetics to make it happen.  

Here’s that story… and a view of the Boston gene editing meeting that runs into a blizzard, an Indian video star, a CAR-T placed before the horse and an over-qualified camera repair guy… (Read on)

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Snapshots of  CRISPR pioneers George Church (Harvard, MIT), Rachel Haurwitz (Caribou Biosciences)  and Jacob Corn (Innovative Genomics Initiative). 

George Church on synthetic biology and MPNs  (Here.)
Jacob Corn “No one’s hiding their stuff.” (Here.)
The Two faces of Rachel Haurwitz (Here.)

lineOf  mice and men… The unsung little heroes on the front lines of health. Ours. (Here.)

News:  CRISPR researchers target incurable blood cancer (Here.) 

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Coming up:  MPN Quarterly Journal to focus on CRISPR Cas9 and gene editing.
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The List and Map of 174 Patient-Recommended Hematologists

The First Six Combined MPNclinics with Index
The Catalog of Articles. ..
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Comments on: "Spring, 2015" (3)

  1. Howard Selinger MD said:

    How does one get the contact information for Crispr clinical trials related to MPN’s?

    • If you’re asking about CRISPR-repaired cells used in either SCT or direct therapeutic intervention, it’s a little early, at least a year or two. But, as we reported, the immediate therapeutic application of CRISPR is in engineering cells, notably modified CAR-T cells used in immunotherapy. There are a host of clinical trials listed at (There’s a short description of timing at, scroll down to “Putting the CAR-T before the CRISPR” paragraph. And the conclusion of the Jacob Corn profile is excellent on this subject.

  2. Thanks so much Zhen once again!

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