New concept for treatment of hemophilia B



CRISPR tool for gene editing will soon become capable of providing hemophilia B patients with help, for it turned out that a transplant of liver cells modified through this method may replace standard injections received by hemophiliac patients.

The team of Salk Institute performed a transplant of liver cells into mice suffering from hemophilia B. It was observed that the therapy of this kind had restored the capability of the organism to produce clots throughout about a year. Due to this, it is hoped that this unique treatment will allow for the replacement of intravenous injections of coagulation factors that are currently applied with regard to any type of such disorders.

Hemophilia B is caused by a defective F9 gene that codes for coagulation factor IX (FIX). The organisms of hemophiliacs produce an insufficient amount of FIX, due to which life-threatening haemorrhages may occur. The currently applied treatment is strenuous, expensive and time-consuming, as it requires receiving injections on a regular basis - even a few times a week. The scientists of Salk had been trying to develop a replacement therapy, while particular injections of RNA coding for the FIX gene would only yield a one week-lasting effect. It was not before the transplant of CRISPR-modified liver cells was performed when new light was shed on the matter. The experiment relied on collecting blood cells of hemophilia B patients and reprogramming them into stem cells. Further on, the gene mutation was removed with the use of CRISPR, while the cells itself were transplanted as liver precursor cells, namely hepatocyte-like ones, or HLCs, into - so far - mice.

Scientists will have to conduct further research prior to the commerciallisation of this solution, while it is the gene therapy Spark Therapeutics SPK-9001 being used for the treatment of hemophilia today. It is centered around the same mutation as the Salk discovery is, while it provides a functional copy of a gene instead of introducing healthy cells into its structure.


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