A trial of the CRISPR/Cas9 gene editing technology has been proposed for use in a human patient. The National Institute of Health Recombinant DNA Advisory Committee (RAC) formed 40 years ago to oversee and create guidelines for the use of recombinant DNA technology in NIH funded studies. It recently made a few amendments to the guidelines which limited submissions of human gene transfer protocols (experiments in which humans are given some kind of nucleic acid like natural or synthetic RNA or DNA) to only exceptional cases. One such case will be discussed at the June 21-22 meeting of the RAC.
The proposal is to use the immune system of a patient to battle cancer cells. White blood cells would be removed from a person suffering from a cancer such as myeloma, melanoma, or sarcoma. The white blood cells would be altered using CRISPR. Upon returning the altered cells to the patient, they will target and attack the tumor cells.
CRISPR has taken the world of molecular biology by storm since its creation in 2012. It allows for the target and editing of genes in a very precise way. It’s a relatively straightforward and low cost tool that has been broadened and improved since its invention. “Researchers in the field of gene transfer are excited by the potential of utilizing CRISPR/Cas9 to repair or delete mutations that are involved in numerous human diseases in less time and at a lower cost than earlier gene editing systems,” Carrie Wolinetz, associate director of science policy at the NIH, commented in a blog post that revealed the submission of the new protocol.
The plan has been submitted for review by the University of Pennsylvania. That university has already had some experience in this area. They were part of developing a therapy in which a patient suffering from a deadly form of leukemia was successfully treated using genetic engineering on the patient’s white blood cells. These are the same cells to be modified in the proposed treatment. Normally they attack bacteria, and just require a few changes so they will go after other cell types, like tumor cells. In this video below, white blood cells of the immune system are seen attacking cancer after being reprogrammed.
There are plenty of risks associated with these kinds of treatments, and with anything so new there could be unintended and unforeseen consequences. “While the application of new gene editing technologies in this field has great potential to improve human health, it is not without concerns,” continued Wolinetz.
The upcoming meeting of the RAC will be available for public viewing online through the NIH videocast
. For more information about the meeting, check the Office of Science Policy website
To learn more about more about CRISPR/Cas9 gene editing technology, watch the video below in which one of the creators of the tool, Jennifer Doudna, a Professor of the Departments of Chemistry and of Molecular and Cell Biology at University of California, Berkeley, and an Investigator of the Howard Hughes Medical Institute, explains it.
Sources: National Institute of Health
, MIT Technology Review