miR126-5P for treating Motor Neuron Disease
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iR126-5P for treating Motor Neuron Disease
Modality: Gene Therapy |
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About the project:
The need:
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.
The novel target:
- miR126 is highly expressed in motor neurons and muscles and is protective against toxic signals.
- We identified that miR126 is decreased in motor neuron axons and muscles of ALS models.
The product:
miR126- RNA nucleic acid-based gene therapy for the treatment of ALS.
miR126- overexpression to protect and restore motor neuron axons, muscle contraction and prevent paralysis in ALS.
The current development:
- miR126 overexpression in diseased neurons extended their axonal growth and preserved muscle contractions in a proprietary microfluidic "lab-on-a-chip" co-culture platform.
- Treating ALS mice with miR126 intramuscular injection rescued muscle denervation
and resolved paralysis of the treated limb. - The mechanisms of miR126 protective effects were elucidated, via inhibition of toxic signals.
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