Scientists have developed more techniques that are classified as gene editing, but not necessarily involving CRISPR itself. In fact, I believe the CRISPR craze has re-energized laboratories and of course funders to take the plunge into the general area and develop even old techniques into viable treatments for truly serious diseases, including Cancer.
There has also been significant discussion around regulation of new techniques since we are talking about very new areas of genetic engineering that we have little experience with. So DARPA, for one, has invested $65 million dollars into a program that should help prevent serious long term issues for people treated via gene editing techniques in the future.
The program has three main technical objectives: to develop processes that allow greater control of genome editing in living systems, to develop countermeasures that protect genome integrity in populations, and to investigate a way to remove engineered genes from living systems.
This is smart because companies in capitalistic systems can take advantage of the quick fix without caring about long term effects on people and on societies. I never prescribe stopping or blocking research, but since science is moving at a breakneck speed, our regulatory bodies do need to be faster and even more proactive.
I talk more about the sociological impact of these technologies and ideas on regulation in the video below:
What's truly amazing about these techniques is that we're entering the realm of personalized treatments for the most part. What is particular about cancer is that it is caused by mutated DNA. Since the mutation can be located in different parts of the cell to cause the uncontrolled division of cells that is the disease, it means that each cancer is in fact somewhat different and unique to the individual and cancer type (both).
Thankfully, gene editing techniques means giving us the ability to target very specific gene sequences to dole out treatment that would work only for a certain individual.
For example, a lab in Boston's Dana-Farber Cancer Institute got very successful cure/remission results with patients by analyzing the patient's individual cancer neoantigens. These are specific proteins on the surface of cancer cells that aren't present in healthy cells. The lab reproduced the neoantigens outside of the patient, and then let the patient's immune system (T-Cells) attack the proteins once injected back into the patient along with immune system activators. That way, the T-Cells would attack their cancer-specific neoantigens in the blood, but also attacking the cancer cells too since they are full of the mutated proteins.
Ok, this is not gene editing, but it was inspired by the idea of personalized treatment.
Another lab had a similar idea, but instead of injecting the body with the neoantigens, they chose to extract some of the patient's T-Cells and edit their genetic code to better attack the patient's specific cancer type, and then re-inject the cells back. This treatment saw an FDA panel approve of this treatment recently (though not FDA approved yet). Credits for the treatment goes to the University of Pennsylvania and Novartis Corp.
Both cancer treatments mentioned above had over 50% test patient recovery rate or better and did not cause any ill effect on the host bodies and no harmful chemicals were needed in the process either.
Gene therapy techniques like this one and others are still being tested on animals, ready for human trials for cancer and also for other terrible diseases like DMD (Duchenne Muscular Dystrophy). We're quickly getting to very nice roads to broad treatments and cures of very nasty health problems, which is very encouraging!
And because labs everywhere have had a great time finding good ways to use gene therapy and associated techniques, it has also encouraged labs to go as far as using gene therapy to cure genetic diseases found at the embryonic level. Yes, that means using gene editing techniques on embryos, potentially still in the womb, thus allowing the newborn baby to grow into an adult and have a healthy life without a potentially deadly genetic disease running around inside his DNA.
Pretty cool stuff and indeed, something that must be studied further and regulations in place to avoid the possible scenario of causing long term harm in trade for a short term good.
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