CRISPR has been the number one celebrity in terms of genetics, and ever since it was developed, numerous studies had been able to push through with it. The most common function of CRISPR was to edit genes and manage mutations of lab-grown cells that are found in diseases. But today, CRISPR once again stunned many experts as it was medically examined to be capable of treating a disease.
CRISPR Trial Successful Against Transthyretin Amyloidosis, Treats the Untreatable
Treating an individual that has a genetic disorder with CRISPR requires a lot of effort. The powerful gene editor is best known for targeting DNA and replenishing the fragments that need to be altered. In a recent study, CRISPR technology was utilized in a drug for treating a specific disease. This drug fused with CRISPR was tested with several human subjects that are diagnosed with what appears to be a congenital disease, reports Science Magazine.
The disease, which was already present on subjects since they were born, causes severe problems with the cardiovascular organs and nerves. Three of the subjects that experience the disease suffer from irregular toxic protein production in their livers.
Transthyretin amyloidosis is the abnormal pattern in protein production in the body's organs and tissues. This disease is tough to handle, but genetic experts were convinced that CRISPR may put our medical solutions on par with transthyretin amyloidosis. In the recent study published in The New England Journal of Medicine entitled "CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis," it was shown that the CRISPR technology puts a medical advantage against the untreatable transthyretin amyloidosis disease. But although the CRISPR's capability to ease symptoms brought by the disease, experts have a strong hope that it will be developed in the near future as a life-long treatment.
Genome Editing on Blindness, COVID-19 with CRISPR Gene Editing
The recent CRISPR study is also an achievement to the ongoing ventures on treatments that rely on protein-building, messenger RNA (mRNA) which naturally produced by our cells. Meanwhile, these mRNAs have a synthetic version that is also utilized to power 2 of the most used COVID-19 vaccine. Moderna co-founder and Karolinska Institute cardiovascular expert Kenneth Chien says that the capabilities of CRISPR and mRNA have finally met.
The objective of the clinical trials conducted with CRISPR is to alter the mutated gene that serves as the root for transthyretin amyloidosis. Deactivating these genes will eliminate the protein build-up located on the heart and nerves that manifest physical affliction. Before the study, patisiran was the only drug that can handle the work. But thanks to the research of Regeneron Pharmaceuticals and Intellia Therapeutics, the convergence of the patisiran and CRISPR technology worked as expected.
The CRISPR treatment was already tested in a previous study in hopes to fix blood disorders. In this case, CRISPR activates fetal hemoglobin to correct sickle cell disease. CRISPR was modified with an extracted blood stem cells and infusing back to the subjects. Further studies regarding the initial CRISPR treatments aim to treat blindness through virus encoding.
Even though it seems challenging to fuse the CRISPR drug with the toxin-producing liver, genetic engineers have found a solution to infuse it with the organ. Many experiments will still be conducted following the transthyretin amyloidosis treatment to fully achieve the perfect DNA repair and deactivating the gene that causes the untreatable disease, reports Nature.
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