Transcriptional reprogramming of motor neurons for ALS
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Unmet Need: Treatment for motor neuron disease including SMA and ALS. Prevents motor neuron death and muscle degeneration. Thus, there remains a great and unmet need for an effective treatment for these motor neuron diseases.
This technology is a gene enhancer, capable of facilitating either overexpression or knockdown of genes in a highly-specific and temporally-controlled manner in cholinergic motor neurons of the central nervous system. A spinal motor neuron-specific enhancer for cell-type specific gene expression control in the developing and adult central nervous system. This technology is gene expression regulatory element that specifically targets cholinergic spinal motor neurons in the central nervous system and can be used for gene expression control throughout the mouse or human lifespan.
Unmet Need: Comprehensive therapeutic for patients with retinal degeneration. While metabolic imbalances and oxidative stress are thought to contribute to disease progression, there are currently no effective, broad-based treatments available for patients with genetic retinal degenerative disorders, including RP, glaucoma, autosomal dominant optic atrophy (ADOA), and age-related macular degeneration. The Technology: Gene therapy for targeting metabolic imbalance and treatment of retinal degeneration.