Calming the destructive cells of ALS by two independent approaches northwesternu
Recombinant adeno-associated virus -based gene therapies offer an immense opportunity for rare diseases, such as amyotrophic lateral sclerosis , which is defined by the loss of the upper and the lower motor neurons. Here, we describe generation, characterization, and utilization of a novel vector system, which enables expression of the active form of hepatocyte growth factor under EF-1α promoter with bovine growth hormone poly sequence and is effective with intrathecal injections.
Therefore, we investigated whether intrathecal delivery of HGF would have an impact on one of the most common pathologies of ALS: the TDP-43 pathology. Increased astrogliosis, microgliosis and progressive upper motor neuron loss are important consequences of ALS in the motor cortex with TDP-43 pathology. We find that cortex can be modulated via intrathecal injection, and that expression of HGF reduces astrogliosis, microgliosis in the motor cortex, and help restore ongoing UMN degeneration.
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