For many people, genetic disorders are a very real, and incurable, problem. Immune disorders like psoriasis and blood borne diseases like sickle cell anemia affect millions around the world. With treatments costing thousands of dollars or more, the results are rarely worth the cost, but they are better than the alternative.
Now, researchers at Stanford University have apparently found a way to make serious inroads in treating the untreatable. Led by Dr. Matthew Porteus, an accelerating research movement is using CRISPR- Cas9, a new technique, to try to cure crippling genetic diseases such as sickle cell anemia and muscular dystrophy.
What is This Compex Gene Therapy?
A Palo Alto native, Porteus, 51, has watched as too many of his patients declined due to complications from blood and immune system diseases as potentially lethal as sickle cell anemia, severe combined immunodeficiency disease and beta thalassemia. Although these are all different diseases, they have one very important thing in common: they are all are caused by one mutation in one gene within a single cell type. Just one.
With CRISPR, you excise the single bad gene, introduce a good gene, and some intensive chemotherapy will kill the remaining damaged cells and make room for new ones, which are then infused into the patient. They have tested and proven their approach with experiments on human cell cultures in mice, and they hope to begin human testing in 2018. The biotech firm, CRISPR Therapeutics of Cambridge, recently received an investment of $335 million from German pharmaceutical giant Bayer AG to form a joint venture to research CRISPR-based treatments.
Unfortunately, while single cell issues are now being treated, many other common conditions with a genetic basis, such as autism, diabetes, or heart disease, involve multiple genes and are currently out of reach of medical science. Porteus and his team’s current strategy is to first focus on a handful of genetic diseases and then expand: “If we prove how to do it, that opens up the possibility for many diseases of the immune system and blood system.”
Stanford University is Leading the pack
Following in their footsteps, scientists at Duke University and two other independent labs are using the same approach to fix a muscle gene for those affected by an until-now incurable type of muscular dystrophy. Boston researchers are similarly using CRISPR to treat a rare, but hereditary, eye disease that often causes blindness. Teams elsewhere are also working on finding and repairing the genes responsible for conditions like Huntington’s disease, Sanfilippo syndrome and cystic fibrosis, among many other disorders.
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