The United Kingdom this week approved the use of the common editing technology Crispr as a treatment for sickle cell anemia, also known as “thalassemia,” and chronic anemia. Regulatory approval is considered a new revolution for medicine.
The method was developed by Nobel Laureates in Chemistry, Jennifer Doudna and Emmanuel Charpentier, and is considered the “single cure” for getting to the cause of these diseases, which was first highlighted. The Wall Street Journal.
Key challenges include the safety of the practice. This is because the patient must undergo chemotherapy and other intensive procedures, including reimplantation of cells in the body, harvesting and manipulation of genetic material “ex vivo”, meaning outside the body. .
For researchers, it would be very interesting to conduct the treatment in an “in vivo” model. In other words, delivering gene editing directly to the patient without extracting and re-introducing the cells.
Expectations for the future
These deadlocks have had an impact on investors, who are increasingly wary of the case. However, pharmaceutical companies Vertex Pharmaceuticals and Crispr Therapeutics are interested in the subject.
In the future, gene therapy may expand to other diseases. In this case, it involves the liver, with other research planned to fight diseases such as cancer, AIDS and Alzheimer’s disease. The technology is awaiting approval from the US regulatory body, the FDA, which is scheduled for December.
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