U.S. CRISPR gene editing trails
Rachel Craighead, Contributing Writer
As the world advances, so do the technologies available to us in the forefront of
medicine. CRISPR, which is a gene editing technology whose name is based on the genetic name
for genetic code found in bacteria and viruses. The name of the technology also helps
communicate how the technology is used; the same aspects of naturally occurring CRISPR
genome and genetic code seek out bacterial and viral cells in order to get rid of them during
treatment. Recently, despite some controversy and public opinions, CRISPR trials have taken
place. CRISPR gene editing trials are currently being performed by the University of
The spokesperson for the University of Pennsylvania in Philadelphia established
that two patients, one suffering from sarcoma, which is a cancerous form of tissue that develops
on the connective tissue of bodies, and another patient who suffers from myeloma, which is a
form of cancer that is based in plasma cells, have both started to receive CRISPR gene editing
treatment for their diseases. These patients fall in line with CRISPR’s early trial treatments, as
most of the CRISPR gene editing technology has been used to treat blood disorders and cancers
based on blood and plasma cells. These are only two of the reported 18 participants in the
CRIPSR gene editing trials. CRIPSR is also used by these patients due to them having late stage
versions of cancers, and often exhausting other treatments or traditional medicinal methods.
CRISPR or CaS9 therapy, which is currently underway for the two patients mentioned above, is
using CRISPR edited T cells in order to attack the cancer cells growing within these individuals.
Although CRIPSR is being used to treat two severe cases of cancer, it is not limited to end-stage
diseases. There are also CRISPR trials underway that are attempting to treat blood borne diseases
such as beta thalassemia and sickle cell. There are several cases in which individuals carry the
sickle cell gene but themselves are not sick however, if they pass this gene on to their offspring
when the other partner has sickle cell, the child is almost guaranteed to have sickle cells.
CRISPR technology aims to end this possibility by using their gene editing to help those not only
with the dormant gene, but additionally to prevent their offspring from inheriting the disease.
CRISPR gene editing caused a buzz a few years ago when two Chinese twin girls were born
using the technology in order to make them healthier and less prone to disease.
With the new implementation of the CRISPR trials here in the US, the company and methods have seen some controversy from groups who protest gene editing and believe that gene editing is wrong, claiming that if it becomes popular, people will attempt to have “Designer babies”. Many also argue about the ethical implications of genetic testing on human subjects. However, even though these trials are underway, they are still many, many years away from being approved as methods for disease prevention and treatment by the FDA. That being said, these trials are a small glimpse into the future of medicine that may end up developing ways to cure thousands of genetic diseases.