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What is CRISPR?
The term CRISPR is short for "clustered regularly interspaced short palindromic repeats." In 2012, scientists studying bacteria discovered that within a bacteria's DNA, there are codes for different sorts of viruses. These codes were created as the bacteria encountered new viruses.
If one of these viruses enters the bacteria again, the bacteria can use an enzyme known as Cas9 to fight the virus by tearing apart its DNA.
What does this mean for medicine? Scientists can use this technology to either remove or replace problems within a person's genome. CRISPR is not the first gene therapy scientists have tried, but the process is much more simple and cost-effective than previous methods. CRISPR gives scientists the ability to better find the specific area of the genome where the mutation occurs.
Once the mutated area is discovered, scientists can do a simple cut and paste within the gene. If tests continue to prove successful, the CRISPR/Cas9 method may be an effective way to fight many of our worst diseases.
What Types of Diseases May Be Cured By CRISPR?
Although there are many experiments and trials ahead, CRISPR has the potential to help scientists treat and even cure almost any disease that is caused by an issue in a person's DNA - potentially up to 89% of these diseases. Once scientists identify the portion of the DNA that causes the disease, it is relatively simple to go in and either change the code or remove the defective section.
Additionally, while there are still many studies being run to determine the possibilities and best uses of CRISPR, scientists are learning much more about diseases during these studies. Even if CRISPR isn't the panacea many are hoping it will be, it is getting us closer to finding cures.
Currently, a few experimental trials are using CRISPR methods for immunotherapy in cancer patients who have had little success with other treatments, such as chemo and radiation. This treatment involves the editing of T cells to help in the attack of the cancer cells. Early tests using these treatments in late-stage cancer patients are showing some success, and doctors are hopeful that as they learn more, they can create even more effective treatments.
There are many other possibilities when it comes to CRISPR and cancer treatment. Researchers can use these techniques in the lab to essentially take apart a cancer cell, gene-by-gene, to discover exactly what is causing the most harm. They can then use this knowledge to engineer effective drugs to target cancer cells.
Scientists also hope to be able to put CRISPR genes directly into a tumor and fight cancer at its source. Early studies have shown that these treatments can slow the rate of growth and make chemotherapy more successful.
HIV / AIDS
We have come a long way in our treatment of HIV and AIDS. In the last decades, scientists have developed antiretroviral therapy that can slow the progress of the virus. Patients now have a much longer life expectancy.
Now, using CRISPR technology, we are closer than ever to curing the virus. Previously, therapy consisted of slowing the disease, but it was not able to completely remove the virus from the cell. CRISPR may allow doctors to completely remove the viral strain.
This treatment has not been tried in human patients, but doctors have had some success with mice whose cells have been "humanized."
People with Cystic Fibrosis (CF) are born with a mutation on a particular gene. This disease can be debilitating and can significantly shorten a person's lifespan. There is currently no cure for CF and treatments do little to slow the disease.
CRISPR offers some new possibilities, though. Doctors could potentially use the technology to get into the genome, find the mutation, and remove it. This has to be done one cell at a time - making it a potentially arduous task.
Treating CF with CRISPR poses several challenges. In particular, many of the cells that are affected by CF are located in parts of the body like the lungs, which are difficult to access. Scientists are working to discover new methods to get around these problems.
Sickle Cell Anemia
Sickle Cell Anemia is a disease that affects the blood. CRISPR technology is showing a lot of promise in treating this disease and other disorders of the blood cells.
Blood disorders are easiest to treat with this technology because the blood can be removed from the body, edited, and then put back in. At least one patient has had this treatment so far, and while it is still early days to make a statement about long-term success, the results are promising.
While CRISPR will not be a cure for all forms of blindness, there is potential for those who are born blind as a result of a genetic mutation. Blindness is actually the first disorder that scientists have tried to treat with CRISPR by putting it directly into the body. The CRISPR gene was put into the patient's eye and is working to correct the mutation that causes blindness.
While the results for this treatment are inconclusive, early results are showing some promise.
CRISPR is showing a lot of promise in the field of gene therapy. After several years of testing on animals, scientists are starting to take the technology into clinical trials. While it may not be a cure for all diseases, it is playing a vital role in the way we understand and treat disease. In years to come, it is likely that CRISPR will have played at least a small part in the discovery of most of our cures.
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