05 June 2018

A single injection of genome-edited hepatocytes might provide a long-term treatment for patients with haemophilia B, according to a new scientific study.

In animal models, the proposed treatment has lasted for up to a year, according to US researchers.

Researchers from Salk University, Pittsburgh, say their findings could drastically change how people with haemophilia B live and could also pave the way for similar treatments for other, related genetic disorders.

Publishing in Cell Reports, the team demonstrated in mice that the blood disorder can be treated for life with one single injection that contains disease-free liver cells that can produce the missing clotting factor IX (FIX).

In the study, blood cells from two people with severe haemophilia B, who are unable to produce FIX, were reprogrammed to become induced pluripotent stem cells (iPSCs), which have the capability to differentiate into many other cell types.

Using genome targetting technique CRISPR/Cas9, they repaired the mutations in each patient's FIX gene and then differentiated the repaired cells hepatocyte-like cells (HLCs), which were transplanted into the livers of genetically engineered mice lacking FIX.

The new HLCs produced enough FIX to enable the mice to form normal blood clots. The cells continued to survive and produce the protein for at least a year after the transplantation.

In the future, transplantation of these edited liver cells may be a feasible long-term treatment for haemophilia patients.

First author Suvasini Ramaswamy said: “The appeal of a cell-based approach is that you minimise the number of treatments that a patient needs. Rather than constant injections, you can do this in one shot."

Source: Ramaswamy, S., Tonnu, N., Menon, T., Lewis, B.M., Green, K.T., Wampler, D., Monahan, P.E. and Verma, I.M., 2018. Autologous and Heterologous Cell Therapy for Hemophilia B toward Functional Restoration of Factor IX. Cell reports23(5), pp.1565-1580.

Link: https://www.cell.com/cell-reports/fulltext/S2211-1247(18)30497-2


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