miR126-5P for treating Motor Neuron Disease

miR126-5P for treating Motor Neuron Disease

iR126-5P for treating Motor Neuron Disease


Modality: Gene Therapy

  • Prof Eran Perlson, PI, Sagol School of Neuroscience and Sackler Faculty of Medicine
  • Dr. Amir Dori, PI, Neuromuscle clinic and laboratory director, Neurology, Sheba Medical Center, Tel-Hashomer, Tel-Aviv University


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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