A new treatment is showing promise against Huntington’s disease in laboratory studies, researchers reported last night.
Laboratory mice responded to a single injection of the therapeutic protein, known as a zinc finger.
The protein is designed to target mutant copies of the Huntingtin gene, suppressing its ability to cause harm.
In the study, reported last night, the injection repressed the gene for six months or more.
The project at Imperial College, London, was backed by EU funding, and involved researchers from Universitat Pompeu Fabra, Spain.
The six months’ worth of effectiveness was achieved by tweaking the ingredients of the zinc finger to camouflage it from the immune system, according to the researchers. In early studies the treatment lasted for just two weeks.
Researcher Dr Mark Isalan said the results were "extremely exciting."
He said more research was needed – but he hoped clinical trials could begin within five years.
According to the research, reported in Molecular Degeneration, 12 mice were involved in the research. The study found that after 24 weeks, some 23% of the gene’s activity was repressed. In the first three weeks 77% of the gene expressed was suppressed.
Dr Isalan said: "While these encouraging results in mice mean that the zinc finger looks like a good candidate to take forward to human trials, we still need to do a lot of work first to answer important questions around the safety of the intervention, whether repeat treatments are effective, whether there might be longer-term side effects, and whether we can extend and increase the benefits beyond six months.
"In this study we weren’t looking at how repressing the gene activity affected the symptoms of the disease and this is obviously a critical question as well. However, we have reason to be confident from our previous studies that repressing the gene does in fact significantly reduce symptoms."
Deimmunization for gene therapy: host matching of synthetic zinc finger constructs enables long-term mutant Huntingtin repression in mice Molecular Neurodegeneration September 2016
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