PYC seems to have begun to build real value in its platform….as it said it would. The importance here is delivery of an enzyme to the nucleus instead of just the cytoplasm (which it had previously done with lactamase and GFP).
Significance: for the cells to go from green to red, the enzyme Cre - which has been delivered by the FPP - has to engineer the DNA in the nucleus of the cell.
Who cares? anyone who is in the business of using genetic engineering enzymes such as CRISPr/Cas9, which includes reagent companies, therapeutic companies, plant and animal biotechnology companies. FPPs could provide a quick, cheap and easy way to deliver these enzymes. And if it can be done in a cell-specific manner --- potentially huge.
It can be applied in both drug discovery and therapeutic treatments. In drug discovery, CRISPR/Cas9 allows us to rapidly screen for the activity of different genes or functional domains of proteins. People are already using it to identify targets in oncology by taking a cancer cell and screening every gene in the genome to see whether it is involved in the cell’s ability to continue dividing.
When it comes to therapeutic applications, CRISPR/Cas9 has the potential to affect dozens of therapeutic areas and hundreds of diseases, both rare and common. It allows us to target the underlying cause of a disease and possibly cure it by modifying the patient’s genome. Some long-term applications are extremely exciting, such as regenerative medicine.
In the near term, CRISPR is likely to be developed and approved for treating monogenic diseases. I’m excited about the potential for treating blood-based disorders like beta thalassemia, which is caused by a mutation in or near a single gene. The standard treatment today is regular blood transfusions, and the only cure is a bone-marrow transplant from a donor, which carries a significant risk of morbidity and mortality. CRISPR/Cas9 offers a safer and more effective treatment where the clinician takes stem cells from the patient, edits them to correct the defect, and then readministers them to the patient.
Beyond healthcare, there’s a plethora of applications. CRISPR/Cas9 can be used to eliminate pests, and microbes can be engineered to produce biofuels more efficiently. In agriculture, the technology can accelerate crop engineering by enabling us to modify genes in plant seeds more quickly and precisely. CRISPR/Cas9 can be used to make corn resistant to herbicide, for example, or to produce grapes that are freeze resistant so that they aren’t damaged by cold storage.
Looking ahead, CRISPR/Cas9 has the potential to drive truly personalized medicine. Medicine has not yet adapted to the genomics revolution that has taken place in the last two decades. What has been missing is a better tool to allow us to interrogate the genomic data and act on it to elicit a therapeutic effect. CRISPR/Cas9 is the first viable tool not only to enable us to explore and interrogate the genome but also to provide us with the drug to repair the genome in diseases where limited treatment options are available for patients today.
One can envision a time in the not-too-distant future when a patient presents with a genetic disease. Her genome is sequenced, and a genome-editing drug is custom made, targeting her specific mutation. The patient is subsequently treated and potentially cured, in a cost-effective manner. The CRISPR/Cas9 technology has the potential to drive a medical revolution in the near future.
PYC Price at posting:
2.8¢ Sentiment: None Disclosure: Not Held
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