ALS is a neurodegenerative disease characterized by the progressive loss of motor neurons — the nerve cells that control movement. ALS is progressive, meaning that symptoms worsen over time. Although there is currently no cure, there are treatments available to slow ALS disease progression and improve quality of life for patients. There are also a number of experimental treatments that are being developed by researchers.
How miQure works
To make a protein, cells first make a temporary copy of the gene that encodes for that protein. This copy is called messenger RNA or mRNA and is used by the cell to make the protein. In ALS, the most common disease-causing mutations occur in a gene called C9orf72. The mutations cause C9orf72 mRNA to build up inside cells in aggregates and sequester proteins, which eventually causes cell death.
miQure contains two very small RNA pieces (called microRNA or miRNA) that are complementary to the mutant C9orf72 mRNA. The miRNAs bind to the mutated mRNA and target it for degradation before it can aggregate.
A non-infectious virus called an adeno-associated virus, which cannot replicate or cause illness, is used to deliver the treatment to the cell.
The results from two preclinical studies about the potential role of miQure in treating ALS were published in the journal Molecular Therapy-Nucleic Acids.
The first study demonstrated that the miRNA contained in miQure reduced levels of mutated C9orf72 mRNA in cells grown in the laboratory. Next, the researchers treated nerve cells (derived from patients) with miQure, which reduced the formation of toxic mRNA aggregates within the cells.
The second study confirmed that miQure is able to reduce the accumulation of mutated C9orf72 mRNA. The authors then validated the treatment in a mouse model of ALS that carries the human form of the disease-causing C9orf72 gene. Following treatment, a reduction in faulty C9orf72 mRNA was found in the mouse brain.
According to uniQure, these findings support pursuing miQure as a therapy for ALS.
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