CRISPRs are taking the scientific community by storm, or so it seems. But what are they? Where did they come from? And why should ophthalmologists care?
Don’t worry – our team at Sightpath Medical is here to fill you in.
What are CRISPRs?
CRISPR-Cas stands for Clustered Regularly interspaced Short Palindromic Repeats associated systems, and they are powerful genome editing tools. They were first discovered in the immune systems of archaea and bacteria. Bacteria must fight off a plethora of viruses. If the bacteria are successful, CRISPRs store some of the invader’s foreign DNA in the form of chopped up sequences which can teach it how to better fight off the same virus in the future.
CRIPSRs are now being isolated and exploited to permanently alter genes of plants, animals, and humans to ensure certain traits and treat genetic diseases.
For a more complete history on CRISPRs, check out this article.
How do CRISPRs work?
To accomplish this technique of gene editing, CRIPSR “spacer” sequences are transcribed into short RNA sequences – the RNA then targets a specific DNA sequence, allowing the CRISPR-Cas protein to create a cut in the targeted DNA. This enables researchers to insert new sequences and edit the gene with acute precision.
In other words, if DNA is like the blueprint to a home, CRISPRs can be thought of as the renovation team hired to gut and rebuild the kitchen you’ve talked about remodeling for years.
CRIPSRs in Ophthalmology
From laser surgeries to nanobiotechnology, the ophthalmology field has never been a stranger to innovation. Recently, CRISPRs have made their way into the spotlight.
So far, there are 250 genes that have been identified in the development of retinal diseases – in other words, researchers know what and where they want to explore CRISPR usage for ophthalmic diseases. Here are some of our favorite studies!
- So far researchers have identified 250 genes in the development of retinal diseases.
- CRISPRs have been used in Zebrafish to create an animal model to research the MFRP protein responsible for a genetic mutation that causes extreme hyperopia.
- Ophthalmic research and therapy testing rely heavily on the use of mice research. CRISPRs have made it more cost-efficient and quicker to edit mice genomes to contain specific mutations that cause ocular diseases.
- CRISPRs are being used to develop various therapeutic treatments that may help restore sight for patients. This process is called CRISPR-mediated gene therapy.
- Researchers have previously been limited to treat recessive inherited disorders, but there’s talk that CRISPRs have the potential to go further and treat more complex, autosomal dominant inherited disorders with the ability to cut out the dominant negative mutation within the DNA sequence.
- CRISPR technology has the potential to help a huge number of patients with inherited retinal dystrophies. Moreover, utilization of these tools doesn’t seem to be slowing down anytime soon.
You will have to keep an eye out for exciting and cutting-edge ophthalmic advancements in the future!