The cusp of science and medicine is seeing all kinds of new potential, thanks to the gene-editing technology of CRISPR-Cas9. Short for Clustered Regularly Interspaced Short Palindromic Repeats, CRISPR is a string of DNA sequences found in bacteria and other organisms.
The sequences originate from viral DNA fragments, which is what contributes to CRISPR's potential as an antiviral and anti-disease tool. Here are some ways in which this technology is leading to new medical breakthroughs and developments.
Leading the Way in Disease Elimination
CRISPR-Cas9 and the use of custom lentivirus, could help with eliminating some of the most deadly diseases on the planet, by enabling researchers to target & modify specific DNA sequences.
For example, malaria, which is carried by mosquitoes, kills over a million people each year, and most of them are children under the age of five. By genetically editing the fertility gene in mosquitoes, scientists are hopeful that they can limit the prevalence of mosquitoes and, therefore, malaria and related diseases.
Another hot topic in the CRISPR-Cas9 field is fighting off Huntington's disease. This is a progressive brain disorder, similar to Alzheimer's, and it affects motor skills, mood, and brain function.
Huntington's is caused by a defective gene, making it a prime candidate for CRISPR-Cas9 research. Scientists have been experimenting with editing out the Huntington's disease gene in the DNA of mice test subjects. The result was a reversal in the progression of Huntington's symptoms, which shows huge promise in how it could positively affect humans with the illness.
Fighting Antibiotic Resistance
Antibiotic resistance is a real problem in today's medical society. This is one of the biggest issues in modern medicine, and there are about two million people in the United States who get infected with an antibiotic-resistance virus each year.
As bacterial viruses develop and assume the ability to resist certain antibiotics, thousands more people die each year. CRISPR researchers took a look at bacteriophage viruses and added antibiotic-resistant gene sequences into the bacteria's DNA.
The result was a sort of triggered self-eliminating mechanism. This means that CRISPR-Cas9 could be a way to drastically reduce the number of antibiotic-resistant bacteria.
Less Expensive Disease Diagnosis
CRISPR-Cas9 is one of the best ways that medical scientists can locate the gene origins of certain diseases. This could make disease diagnosis quicker and less expensive in the future. Researchers can look at a person's blood, urine, or saliva and study it to see if there are any genetic markers of a pathogen.
Cutting out or deleting that specific sequence of DNA can eliminate the prevalence of certain diseases. One such example is Duchenne Muscular Dystrophy. Scientists know the gene that causes this disease, so they can identify that specific mutation, cut it out, and repair it.
Changing the Idea of Organ Donation
Another medical tactic that CRISPR-Cas9 is taking on is organ donation. The Harvard Medical School recently used CRISPR technology to edit not one, but 62 genes in a pig cell simultaneously. The pig genome includes fragments of an antiquated viral infection, genes from the porcine endogenous retrovirus.
Scientists have been able to use CRISPR technology to eliminate the gene in pigs, which primes the creatures to be organ donors for humans in need. In one experiment, researchers implanted pancreases from pigs free of the retrovirus genes into humans with diabetes.
The humans who received the new pancreases showed no signs of having the retrovirus, which was promising. Scientists will have to keep experimenting with this, including using larger organs to see if any new risks arise.
Clearly, there is a lot going on in the field of CRISPR-Cas9 right now. While more research is needed, there is great potential here, both in terms of medical solutions and scientific advancement.
