Perlson and his team uncovered a novel mechanism that contributes to neurodegeneration that takes place in ALS models. Using both in vitro and in vivo ALS models, we demonstrated that alterations in miR126-5p, which regulates the secretion of muscle-toxic factors such as Sema3A, and its axonal receptors, facilitates a non-cell-autonomous mechanism underlying axon/NMJ degeneration.

UNMET NEED

Finding efficient treatment for ALS has not yet been achieved and is an unmet and urgent medical need.
OUR SOLUTION
Novel treatment for motor neuron disease, spinal cord and peripheral neuron injuries by overexpression of mIR126 using virus constructs

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

secreting toxic factors such as Sema3A. (B) Downregulation of miR126-5p in diseased MN's increase the levels of caspase 3 and causes shorter neurites and impaired cell survival. (C) CRMP4 expression levels is elevated in the MNs’, and activation of intrinsic mechanisms of action facilitate a retrograde death signal. Using virus constructs we will over express mIR126-5p to treat ALS.

OUR PRODUCT
Over expression of mIR126 as gene therapy approach

DIFFERENTIATION
No such approach

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