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    Novel rAAV vector mediated intrathecal HGF delivery has an impact on neuroimmune modulation in the ALS motor cortex with TDP-43 pathology

    Recombinant adeno-associated virus (rAAV)-based gene therapies offer an immense opportunity for rare diseases, such as amyotrophic lateral sclerosis (ALS), which is defined by the loss of the upper and the low...

    Barış Genç, Boram Nho, Hana Seung, Benjamin Helmold, Huiwon Park… in Gene Therapy (2023)