A single injection of a new treatment has reduced the activity of the gene responsible for Huntington's disease for several months in a trial in mice.
Huntington's disease usually only begins to show symptoms in adulthood. There is currently no cure and no way to slow the progression of the disease; symptoms typically progress over 10-25 years until the person eventually dies.
Now, researchers have engineered a therapeutic protein called a 'zinc finger'.
Huntington's disease is caused by a mutant form of a single gene called Huntingtin. The zinc finger protein works by targeting the mutant copies of the Huntingtin gene, repressing its ability to express and create harmful proteins.
In the new study involving mice, published in the journal Molecular Neurodegeneration, the injection of zinc finger repressed the mutant copies of the gene for at least six months.
In a previous study in mice, the team had curbed the mutant gene's activity for just a couple of weeks. By tweaking the ingredients of the zinc finger in the new study they were able to extend its effects to several months, repressing the disease gene over that period without seeing any harmful side effects. This involved making the zinc finger as invisible to the immune system as possible.
Paper: “Deimmunization for gene therapy: host matching of synthetic zinc finger constructs enables long-term mutant Huntingtin repression in mice”
Reprinted from materials provided by Imperial College London.