Antisense Morpholino Treatment to Increase SMN Levels and Treat Spinal Muscular Atrophy

A treatment for spinal muscular atrophy.

The Need

Spinal Muscular Atrophy (SMA) is caused by deletion or mutation of SMN1 and retention of SMN2, this in turn leads to reduced levels of the full SMN protein. SMA is the second leading cause of neuromuscular disease, and many people living with SMA need round-the-clock care from their families and also from a variety of medical specialists. This invention provides a treatment for SMA.

The Technology

Ohio State University researchers, led by Dr. Arthur Burghes, have developed a treatment for spinal muscular atrophy (SMA) that involves an intrathecal (the space between the protective membrane covering the spinal cord and the spinal cord itself) injection of antisense morpholinos (ASO). The use of ASO's delivered to SMA patients in particular aims to correct the reduced level of SMN in SMA patients. The ASO's bind regions of the SMN2 gene in order to increase the production of full length SMN mRNA and thus SMN protein.

Commercial Applications

  • Spinal muscular atrophy treatment

Benefits/Advantages

  • No marked toxicity in any dose given in animal trials
  • Compares well with other ASO treatments for SMA
  • Increased weight in animal trials
  • ASO can be administered both intracerebroventricularly and systemically, resulting in correction of SMN in tissues outside the nervous system

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