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“If I hadn’t done this functional study, where, knocking out the PLK4 gene, resulted in the inhibition of cell proliferation I wouldn’t have discovered that PLK4 is essential for AT/RT growth. PLK4 is only slightly elevated in these tumors. However, this gene is tightly regulated and slight increases in its expression result in an aggressive tumor phenotype. This overexpression can be targeted by inhibitors opening a new therapeutic prospective for children with AT/RT. Significantly, we also found it elevated in other embryonal tumors of the brain, what may have a larger impact in patient care … the fact that we used the Invitrogen LentiArray Human Kinase CRISPR Library for the functional assay was key.”
Simone T. Sredni , MD, PhD,
Associate Professor of Pediatric Neurosurgery
Ann and Robert H. Lurie Children’s Hospital of Chicago/Northwestern University Feinberg School of Medicine
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Olivier Humbert, PhD
Staff Scientist in Hans-Peter Kiem’s laboratory
Fred Hutchinson Cancer Research Center
Curiosity about how my surroundings and living things work; I love the excitement of carrying out experiments, the anticipation for results, and the fulfillment of knowing that I am contributing to the development of new medical treatments.
Knowing that I am working with cutting-edge technologies to develop therapies that will become available to patients in the next 5 to 10 years.
Our goal is to treat hereditary diseases by correcting the underlying mutation in bone marrow stem cells so that these cells will be curative after they are transplanted back to the affected patient. The correction of mutations can be done by means of non-infectious viral vectors or more recently with nucleases that target specific sites in DNA, also known as molecular scissors (such as CRISPR-Cas9). Our laboratory covers a wide range of research, spanning basic science all the way to clinical studies; examples of diseases we work on are blood disorders (sickle cell anemia), severe combined immunodeficiency, and Fanconi anemia (defects in DNA repair).
One challenge is to correct mutations and engineer DNA in bone marrow stem cells with high efficiency and without affecting the identity of these cells so that they will differentiate into all blood cell types and reconstitute the hematopoietic system of a patient after transplantation. Another big challenge is in the scale-up, where we are looking to engineer the DNA of several hundred millions of cells obtained from patients.
One solution is to find a nuclease platform (such as CRISPR-Cas9) and nuclease delivery method (such as electroporation) that can treat large numbers of cells with minimum toxicity and high efficiency so that they will be therapeutic after they repopulate the patient. Another solution is to refine our definition of true, long-term hematopoietic stem cells, which will decrease the number of these cells that need to be treated.
We have so far done two transplant experiments in a preclinical animal model aimed at treating hemoglobinopathies (blood disorders). The results are very encouraging, but we want to follow up on our treatment for over a year to really understand how effective and safe our therapy is.
Current gene editing technologies allow us to target virtually any site in the genome in pretty much any cell type. In addition, the improved efficacy of TrueCut Cas9 Protein v2 allows us to reduce the amount used and minimize toxicity.
Improved efficacy and safety of technology equals more potential to bring an effective therapy to the patient. These reagents are also available in large-scale [quantities] and in cGMP-grade for easy translation to the clinic.
I am lucky to be working in such an exciting field with lots of potential to cure a variety of diseases. Nevertheless, researchers need to be vigilant to take all necessary steps to not move this approach too quickly to the patient to avoid any major setbacks.
For Research Use Only. Not for use in diagnostic procedures.
