A novel RNA treatment for ALS

Amyotrophic Lateral Sclerosis (ALS) is a fatal adult-onset neurodegenerative disease with no available treatment. Perlson and his team have identified miR126-5p as a novel target to treat ALS. They have demonstrated both in vitro and in vivo beneficial effects of over expression mir126 on ALS models. They further established the mechanism of action, and optimized AAV virus delivery approach.
ALS is a degenerative fatal disease of the motor nerves. The lifetime risk to develop ALS is about 1:400. There is currently no effective treatment for this disease thus urgent drug development is needed.

Mechanistic-targeting treatment of gene therapy for ALS based on overexpression of miR126-5p  

miR-126 5p beneficial effect in ALS disease. A) In a non-cell autonomous process, muscle and MN communication is alter in ALS disease. Downregulation of miR126-5p promote axon degeneration, NMJ disruption and cell death by regulating muscle

• miR126 overexpression in diseased neurons extended their survival, axonal growth and preserved muscle contractions in a proprietary microfluidic “lab-on-a-chip” co-culture platform.
• Treating ALS mice with miR126 improved muscle denervation, neuron survival and behavioral motor activity 
• The mechanisms of miR126 protective effects were elucidated, via inhibition of toxic signals and dissolving TDP43 mislocalization and aggregation.

miR126-5p FOR TREATING MOTOR NEURON DISEASES PCT/IL2019/050545 National phase in USA and Europe.

miR126-5p Downregulation Facilitates Axon Degeneration and NMJ Disruption via a Non-Cell-Autonomous Mechanism in ALS. Maimon R, Ionescu A, Bonnie A, Sweetat S, Wald-Altman S, Inbar S, Gradus T, Trotti D, Weil M, Behar O, Perlson E. J Neurosci. 2018 Jun 13;38(24):5478-5494. 
Muscle secretion of toxic factors, regulated by miR126-5p, facilitates motor neuron degeneration in amyotrophic lateral sclerosis. Maimon R, Perlson E. Neural Regen Res. 2019 Jun;14(6):969-970.


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