Therapeutically Relevant Engraftment of a CRISPR-Cas9–Edited HSC-Enriched Population with HbF Reactivation in Nonhuman Primates
This week we profile a recent publication in Science Translational Medicine from Dr. Olivier
Humbert (pictured) and Dr. Stefan Radtke in the laboratory of Dr. Hans-Peter Kiem at Fred Hutch.
Can you provide a brief overview of your lab’s current research focus?
The Kiem Lab studies cell and gene therapy with a particular interested in the biology of blood and marrow stem cells and the development and use of novel genome editing technologies. The overall goal is to develop better stem cell transplant and cell and gene therapy treatments for patients with genetic and infectious diseases and cancer.
What is the significance of the findings in this publication?
The CRISPR/Cas9 technology offers many opportunities to treat genetic and infectious diseases, but safety of this technology needs to be validated before it can be used to treat human patients. In our research paper, we modify blood stem cells with CRISPR/Cas9 and demonstrate that these cells can safely persist at high frequencies for over a year to provide therapeutic benefits for hemoglobinopathies. We also show that the treatment of a much-reduced number of CD90 positive stem cells is sufficient to achieve comparable results, thus making this approach more accessible and affordable.
What are the next steps for this research?
We are continuing to monitor results from our experiments to make sure this approach is safe to treat patients. We are also conducting additional experiments to further improve on the therapeutic benefits brought by this method. Clinical trials are very expensive and we are looking for partner companies to move this research to the clinic.
This work was funded by:
This work was supported by grant R01 HL136135 from the NIH