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Gene Editing: The Future of Precision Medicine

  • 3/25/2019 6:30:00 AM
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Gene Editing: The Future of Precision Medicine

John Deaver, MS

            Gene editing has made headlines in recent years as a promising new technology opening new avenues in research, as well as precision medicine.

            The ability for researchers and physicians to perform genetic editing has been around for decades, but historically, it has been an extremely slow and expensive process. One of the reasons for this is that they all rely on using proteins called Nucleases to bind to a specific part of a gene and make a precise cut across the double stranded DNA.  This is an extremely complicated process, and relies on engineering a new protein that will be perfectly specific to a single location within a gene.  This is time intensive, and often relies heavily on computer modeling to predict the activity of the engineered nuclease.  As a result, the process of gene editing is a very time consuming and expensive endeavor that can have unintended consequences.  However, with the discovery of CRISPR, genetic editing has taken large strides forward in terms of reliability, specificity, and ease of use.  The CRISPR system, more accurately known as the CRISPR/Cas9 system, also relies on a special nuclease protein.  However, instead of engineering the protein to bind to the chemically dissimilar DNA, the CRISPR system uses a Cas9 nuclease that contains a small strand of RNA that is a perfect match for the targeted DNA sequence.  In short, this means that creating a new CRISPR/Cas9 system to target any sequence of DNA, in any organism, can take days to weeks, rather than months to years it would take in designing entirely new nuclease proteins.

            So far, this new ability has shown promise in halting the progression of Duchenne’s muscular dystrophy in dogs, by restoring the amount of dystrophin in muscle and heart tissue by up to 92%. It has been estimated that an increase of 15% is needed to significantly help patients.  CRISPR/Cas9 is also being used in clinical trials to treat various types of cancer by removing white blood cells from the patient, modifying them to attack the cancer cells, and then administering them back to the patient.  Early research in animal models suggest that therapies based on CRISPR/Cas9 gene editing could have the potential to treat a wide range of diseases including sickle cell, cystic fibrosis, Huntington’s, cardiovascular disease, and many, many more.  In addition, it shows great promise to be able to reduce the spread of Malaria by modifying the mosquitos that carry the disease.  CRISPR/Cas9 has a fantastic potential to improve treatment strategies for many diseases, and create new strategies for diseases that did not have viable options previously.

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