Could Genome Editing Be a Future Treatment for Hemophilia?
An experimental form of genome editing called clustered regularly interspaced short palindromic repeats or CRISPR/Cas9 could offer a faster and cheaper method of treating people with hemophilia, compared to the current DNA-editing method. Because hemophilia is due to DNA mutations, the genome editing could be used to efficiently slice at the DNA, removing or adding DNA to correct the mutation.
Hemophilia is a hereditary bleeding disorder that stops the blood from clotting normally. The primary symptom is uncontrolled, often spontaneous bleeding in different areas of the body. According to data from the National Hemophilia Foundation in 2020, Hemophilia A happens in 1 in 5,000 live male births. Hemophilia A is about four times as common as hemophilia B. The number of people with hemophilia in the United States is anticipated to be around 20,000 individuals. Moreover, the incidence of hemophilia is not known across the globe but projected at above 400,000 people. Almost 75% of people with hemophilia worldwide are either undiagnosed or receive disparate treatment.
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In Italy, according to the data of the National Center for Biotechnology Information (NCBI) in 2017, the number of registered people with bleeding disorders increased from about 7000 in 2000 to around 8500 in 2011 and more than 11,000 in 2015. The trend is due to an upsurge in the number of patients who are recorded, mainly in those with vWD type 1, mild hemophilia, or other factor deficiencies.
A wide variety of hemophilia treatment is available as adjunctive measures to improve hemophilia if residual bleeding persists despite the correct application of conventional methods for hemorrhage control. Plasma-derived coagulation factor hemophilia treatment is considered to be active agents that participate in the coagulation cascade to form fibrin clots and are effective to make an intact coagulation system. Plasma is the liquid part of blood. It is pale yellow or straw-colored with proteins such as antibodies, albumin, and coagulation factors.
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