There is need for corticospinal motor neuron replacement in various therapeutic fields:
Post-stroke rehabilitation: while there is a number of acute-phase stroke treatments, there are currently no therapies available for patients with a stable and fixed neurological deficit following stroke. Post-stroke rehabilitation could be functionally improved by corticospinal motor neuron regeneration.
In neurodegenerative diseases, such as amyotrophic lateral sclerosis (ALS; aka Lou Gehrig's disease), corticospinal motor neurons are part of the most affected neurons.
HOW IT WORKS
The technology is a method to induce cell transformation into corticospinal motor neurons in vivo.
The method comprises the use of vectors encoding for the genes of interest, thereby opening the possibility of an autologous ex vivo gene therapy approach. Nevertheless, other cell therapy approaches may also be possible.
The expression of the specific genes improves three times the yield of reprogrammed cells into corticospinal motor neurons compared to the actual gene technology.
Fig: In utero electroporation with vectors expressing the genes of interest + GFP into upper layer neurons of the mouse cerebral cortex. At postnatal stages (here, day 14), the GFP-electroporated cells (green) express corticospinal neuron markers and extend their axons towards the spinal cord.
KEY BENEFITS vs. STATE OF THE ART
Up to 90% of targeted neural cells can be reprogrammed in motor neurons: improved cell production, time and cost
The process takes takes just a few hours, as soon as the exogenous genes are correctly expressed
Work with a high quality academic team specialized in neocortical development. A recent publication in eLIFe, previous publications in Nature Communications and Cell Mol Life Sci in 2014, Science in 2013, Development in 2013 and 2011, Journal of Neuroscience in 2011, 2010, 2008, PNAS in 2010 and Nature Neuroscience in 2007.
In vivo proof of concept by in utero electroporation on healthy mice.
Ongoing in vitro reprogramming experiments in astrocytes and fibroblasts.
Regenerative neuronal cell therapy:
After stroke as part of the functional rehabilitation process, by re-introducing corticospinal motor neurons.
Neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS).