Use of Gene Product to Treat Nerve Injury and Sarcopenia

A novel method using the overexpression of survival motor neuron protein to improve motor unity synaptic repair and restore/protect muscle size and function due to sarcopenia

The Need

Sarcopenia, the age-related wasting and loss of strength, is an important neuromuscular problem of aging. It affects up to 50% of individuals by the 8th decade, and can lead to impaired mobility, loss of independence, and increased risk of mortality. The neuromuscular system is comprised of groups of muscle fibers innervated by a single alpha motor neuron, motor axon, and synapses, termed a motor unit. The normal development and maintenance of the motor unit is dependent on trophic interactions between muscle and motor neurons. The presence of a complete complement of intact motor units decreases with age, an important factor in sarcopenia. Although beneficial effects of exercise on sarcopenia have been noted, there are no proven therapies available. Understanding of the factors driving the degenerative changes in aging-related muscle wasting and weakness remains incomplete.

The Technology

The Ohio State University researchers led by Dr. Arthur Burghes and Dr. David Arnold have refined minimally invasive in vivo techniques for the treatment of nerve injury and sarcopenia. The developed techniques utilize the overexpression of survival motor neuron (SMN) proteins that are critical for repair of motor unit connectivity after injury and have the potential to improve nerve regeneration and reduce aging-related motor unit losses.

Commercial Applications

  • Treatment of sarcopenia
  • Treatment of motor nerve injury

Benefits/Advantages

  • Novel target for treatment of sarcopenia.
  • The techniques allow assessment of the whole motor unit in a longitudinal fashion, and can provide important insight into the functional interaction of muscle, neuron, and synaptic factors in the development of sarcopenia.
  • An increased level of the SMN protein increases neuronal repair and protects against sarcopenia. Antisense oligonucleotides, scAAV9-SMN and drug compounds can all be used to increase SMN and are either in clinical trials or clinical use.

Patents

Patent # Title Country
10,357,543 Methods and Compositions for Treating Disorders and Diseases Using Survival Motor Neuron (SMN) Protein United States of America

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