MiR-134 RNA Treatment Reduces Seizures in Mice
In a groundbreaking study published in Molecular Therapy Nucleic Acids, a team of scientists led by Prof. Dr. Andreas B. Vielreicher in Ireland have discovered a potential new approach for treating Angelman syndrome, a rare genetic disorder characterised by seizures, developmental delays, and speech impairments.
The study focuses on a small RNA molecule called miR-134, which has a specific binding spot in the genetic sequence of the UBE3A gene, which is responsible for the development of Angelman syndrome when mutated or varianted. Prior research has shown that reducing miR-134 levels in mice could prevent seizures, a key symptom of Angelman syndrome.
The researchers suggest a mechanistic link between miR-134 and the molecular pathogenesis (disease development) of Angelman syndrome. They found that lowering the levels of miR-134 was effective in protecting against seizures in a model of Angelman syndrome for both young and older mice.
AntimiR treatment, a molecule that blocks miR-134 activity, was shown to protect against sound-induced seizures in another mouse model, with some mice experiencing no seizures at all and others showing reduced seizure severity compared to untreated mice. The anti-seizure effects of silencing miR-134 were observed in both young and older mice with Angelman syndrome, indicating the potential effectiveness of this approach across different age groups.
In addition to its anti-seizure effects, antimiR treatment was found to normalise scores on the open field test in one mouse model, suggesting potential alleviation of anxiety and exploratory behavior issues in Angelman syndrome.
The team conducted a series of experiments in mouse models of Angelman syndrome to explore the potential therapeutic approach of targeting miR-134. They demonstrated that antimiR treatment could lower miR-134 levels in nerve cells derived from a patient, serving as a proof-of-concept for the use of this approach in people with Angelman syndrome.
Targeting miR-134 does not seem to be age-restricted, offering a potential advantage over current gene therapy rescue approaches for Angelman syndrome, which have not been effective for controlling seizures in older mice with Angelman, likely due to the gene's importance during certain stages of development.
miRNAs, or microRNAs, are a separate class of small RNA molecules that can bind to mRNA molecules with specific sequences, preventing them from being used to make protein. By understanding the role of miR-134 in Angelman syndrome, the researchers hope to open up new avenues for treatment and improve the lives of those affected by this debilitating disorder.
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