TREATMENT OF NEUROMUSCULAR DISEASES VIA GENE THERAPY THAT EXPRESSES KLOTHO PROTEIN

Abstract

The present disclosure provides gene constructs containing a nucleic acid sequences encoding a mammalian s-KL, operatively linked to a muscle cell-specific promoter, for use in the treatment of motor impairment that may manifest, for example, in a neuromuscular disorder or disease, utilizing viral and non-viral vectors with muscle cell and motor neuron tropisms containing gene constructs containing a nucleic acid sequence encoding a mammalian s-KL, operatively linked to a promoter such as a muscle cell-specific promoter, is delivered in pharmaceutical compositions containing the expression vector, isolated cells containing the expression vector, and methods of treating motor impairment and motor neuron diseases.

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