16 July 2018

A new novel stem cell gene therapy has been developed that could treat children with Hunter syndrome, British researchers have announced.

The inherited genetic disease (also known as Mucopolysaccharidosis type II, or MPS II) is caused by deficiency of the enzyme iduronate‐2‐sulfatase (IDS) and leads to skeletal abnormalities and cardiorespiratory problems. In severe cases, patients die before adulthood. 

The current enzyme therapy costs more than £150,000 a year per patient and does not penetrate the brain, which is affected in two thirds of patients.

Now a research team at The University of Manchester, UK, has developed a novel stem cell gene therapy that replaces the missing gene in the bone marrow of affected children.

To make this more effective, the team also added a tag to the IDS enzyme, which allows it to pass into the brain.

So far the treatment has been tested in a mouse model.

The researchers say: “Brain-targeted hematopoietic stem cell gene therapy provides a promising therapy for MPS II patients.”

Professor Brian Bigger, who led the research, said: “We expected the stem cell gene therapy approach to deliver IDS enzyme to the brain, as we have shown previously for another disease: Sanfilippo types A and B, but we were really surprised to discover how much better the tag made the therapy in the brain.

“It turns out that the tag didn’t only improve enzyme uptake across the blood brain barrier, but also improved uptake of the enzyme into cells and it appeared to be more stable in the bloodstream - all improvements on current technology.”

The team revealed that the treatment was able to correct the effects of the disease in the bone, joints and brains of affected mice in EMBO Molecular Medicine.

Source: Brain-targeted stem cell gene therapy corrects mucopolysaccharidosis type II via multiple mechanisms EMBO Molecular Medicine 8 June 2018; doi: 10.15252/emmm.201708730

Link: http://embomolmed.embopress.org/content/10/7/e8730


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