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CRISPR-Edited Stem Cells Yield Impressive Early Results

December 12, 2019

doctors looking at x rays

Two patients with serious conditions have shown remarkable results after receiving cutting-edge treatment using CRISPR, the revolutionary gene-editing tool, to alter blood-producing stem cells.

Four months after a cell therapy procedure, a sickle cell disease patient was free of vaso-occlusive crises (blood cells getting stuck in vessels, causing severe pain and potentially organ damage). She’d previously experienced about seven such episodes per year.


In the other case, a beta thalassemia patient was transfusion-independent nine months after treatment. She’d previously needed about 16 transfusions per year.

The goal: pinpointing and ‘repairing’ genetic mutations

Genetic mutations are at the root of both diseases. As STAT explains, research has shown the conditions might be cured if a form of hemoglobin that the body usually stops making soon after birth can be reactivated: “That’s what [the new treatment] CTX001 aims to do: use CRISPR to disable a DNA brake on the production of fetal hemoglobin, thereby giving the body a healthy supply of the vital molecule.”

At the nine-month mark, 99.8 % of the beta thalassemia patient’s hemoglobin was the fetal type, meaning the treatment had worked. In the sickle cell disease patient, 47% was fetal hemoglobin. It’s thought that a fetal hemoglobin level of 25%-30% is sufficient to “cure” a patient with sickle cell disease, according to STAT.

The treatments reportedly involve drawing blood from the patients, isolating hematopoietic (blood-producing) stem cells, and infusing the CRISPR’ed cells into the bloodstream.

“These results are remarkable because they represent the first clinical evidence that CRISPR-Cas9 has real curative potential, Vertex chief executive Jeff Leiden told STAT. “From a scientific and medical validation standpoint, the data are important.”

Larger trials are planned soon.

Hematopoietic stem and immune cells for research

This work is an exciting example of innovative biomedical research involving hematopoietic stem and immune cells, which are available from both healthy and diseased patients through the iSpecimen Marketplace.

We have assembled one of the industry’s largest, most integrated networks of donors and specimen collection sites for these cells. Through the iSpecimen Marketplace, researchers can efficiently procure bone marrow aspirate, peripheral blood mononuclear cells (normal and mobilized), and whole blood – all with corresponding donor profile/clinical data from donors.

Researchers use these types of tissue and cell products to develop stem cell therapies, immunotherapies, vaccines, diagnostics, new treatments for infectious and autoimmune diseases, and in cell-based assays to advance drug discovery/preclinical development.

This work provides hope – and someday perhaps, cures – for very sick patients.

Learn about the iSpecimen Marketplace where you can browse millions of richly annotated, de-identified human tissue and biofluid biospecimens, in addition to hematopoietic and immune cell products. You can join for free and creating a login is easy. Request a quote or custom collection today.

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