Novel Mouse Model for Developing Therapeutic Drugs Against Muscle Atrophy

Mouse model stably overexpressing Ant-1 for muscle atrophy therapy development. 

Background:

Muscle disorders are commonly manifested in mitochondria-induced diseases, which are clinically referred to as mitochondrial myopathies. In classic mitochondrial myopathies, caused by mutations in mitochondrial DNA or in nuclear genes encoding mitochondrial function, biochemical and histological biomarkers indicative of bioenergetic defects can be readily detected. In contrast, the role of mitochondria in other progressive muscle diseases, including sarcopenia is less clear.

Technology Overview:  

Overexpression of the muscle/heart isoform of adenine nucleotide translocase, Ant1 is associated with the clinically well-defined progressive muscle disorder Facioscapulohumeral Dystrophy (FSHD). Upstate Medical University researchers have generated a transmissible transgenic mouse line that stably overexpresses Ant1, resulting in manifestation of severe muscle disorders in the transgenic animals.

Thus, this invention provides a unique animal model for studying the pathogenic mechanism of FSHD and other mitochondria-induced diseases including sarcopenia of aging (aging-dependent muscle wasting), as well as a tool for the development of therapeutic drugs in the treatment of these diseases  

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Advantages:  

  • Ant1-transgenic mice progressively lose muscle, making them relevant for musculoskeletal disorders.
  • Animal lifespan remains unaffected, enabling the detailed study of muscle wasting mechanism

 

Applications:  

  • Research tool for mitochondrial disorders
  • Drug screening for neuromuscular degenerative disorders
  • Muscle degeneration research

 

Patent Information: