CRISPR: The Future of Personalized Medications
The healing of many by genome editing
By Ava Fuhr
Intro
Imagine this: ever since you were a child, you have had blockages in your blood flow. Now, this might not seem like a big deal to you, but just wait. These constantly pain you, make you rushed to the hospital on a regular basis, and get horrible infections quickly.
This, believe it or not, was the life of 37-year-old mother Victoria Gray (pictured above), along with the 250,000,000 other people worldwide suffering from sickle cell disease. Then, in 2019, she got experimental CRISPR injected into her bloodstream. This has changed her life in a way that she does not suffer from sickle cell anymore.
Present
About one in every twelve babies are born with a rare genetic disease. Using their individual phenotypes, scientists can quickly make personalized medication for the specific child’s condition. There are even new cancer therapies that use direct manipulation of the cell’s genetic information.
This main new strategy of custom editing genomes could help thousands, even millions, of people suffering through genetic ‘typos’. A problem with this is that these new therapies are very expensive, at about 10,000 dollars per month! With this cost, pharmaceutical companies currently have no interest in buying or making these drugs. Insurance won’t even pay for them yet! Only a few people have been able to receive this treatment, which is a sign that this project is still in its youth.
An important part of this treatment is called ‘antisense’. This is a tech that, basically, blocks certain RNA from leaving the nucleus with genetic information. This means it could block or silence that gene completely. If used correctly, this means that you could use antisense in personalized meds to completely mute specific bad genes causing genetic disorders.
CRISPR
CRISPR is important in all of these aforementioned therapies because it makes the treatment process even more simple. Just like in any other CRISPR gene editing tool, personalized meds use DNA to specify between species, or even people. How CRISPR works is as follows. CRISPR sequences are transferred onto RNA sequences that guide the CRISPR to the matching DNA strand. When found, the Cas-9 enzyme produced by the CRISPR protein attaches to the DNA and slits it from the rest of the sequence. Doing so, this shuts off the gene associated with that DNA strand. This means that if there was a gene error, the CRISPR protein could shut off that gene and fix the genetic error.
Personalized medication and CRISPR are still fairly new sciences and have much more improvement left in the future. There is so much promise in hyper-personalized drugs, because they have a good chance to help people with rare genetic diseases. A way that precision medicine could change in the future is if geneticists incorporate AI into the diagnosing process. This could make it even easier if AI was able to help people figure out exactly which part of the strand they need to cut away with CRISPR personalized medicine. Another improvement could be using AI to make an early diagnosis of the disease, for early treatment.
Prime Editing
A person who is working on developing precision medicine is David R. Liu (picture below). He works as a professor at Harvard, at the Broad Institute, and at HHMI. He is specifically working on prime editing, which helps edit even more precisely. Prime editing is important because its specificity can help correct genetic mutations causing diseases. This is very new and will hopefully be incorporated into precision medicine.
Prime editing is an important new development in the CRISPR realm. This deals with adding to and taking away from genomes. This is important because it is the newest, easiest technology to replace double stranded breaks or mistakes in the genome.
Conclusion
These new technologies will be helpful to change our lives. With this technology, we can eradicate genetic disorders possibly even before the child is born. And, we can help more people like Victoria Gray, who suffer with genetic disorders and diseases.
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