Research Spotlight: Degenerative Myelopathy


What is Degenerative Myelopathy?

  • Degenerative myelopathy (DM) is a devastating neurodegenerative disease that affects multiple breeds of dog.
  • DM is an adult-onset disease that manifests at the later stages of life.
  • It is characterized by progressive weakness and inability to control hindlimbs, ultimately leading to involvement of forelimbs and complete paralysis.

Why study it?

  • With no current treatments available, euthanasia is the only option available for DM-affected dogs.
  • Recent studies have identified mutation in the Superoxide dismutase 1 (SOD1) gene to be a high risk factor associated with canine DM. In humans, mutations in the same SOD1 gene cause Amyotrophic Lateral Sclerosis (ALS) or Lou Gehrig's disease, a neurodegenerative disorder very similar to canine DM.
  • Therapeutic approaches to reduce the expression of mutant SOD1 in DM-affected dogs may improve survival and preserve neurologic function.


A research study (2165) funded by the AKC Canine Health Foundation and the American Boxer Charitable Foundation has led to a recent report showing a promising biomarker for dogs with nervous system diseases. Similar biomarkers are being studied in ALS patients and findings from this study may also impact human treatments. 

CHF-Funded Study

02210:  Gene Therapy for Canine Degenerative Myelopathy
Principal Investigator: Dr. Brian K Kaspar, PhD; The Research Institute at Nationwide Children's Hospital
Total Grant Amount:  $50,000
Grant Period:  1/1/2016 - 12/31/2018

What is the focus of this study?

  • In this study, a viral-based gene therapy approach to treat DM will be evaluated, utilizing Adeno-associated Virus 9 (AAV9) mediated delivery of shRNA to reduce the mutant SOD1 in DM affected dogs.
  • AAV9 is a safe, well tolerated and widely used vector for gene therapy in animals as well as for humans.

How will this study help dogs? 

If successful, this one-time treatment with AAV9 SOD1 shRNA will result in improved quality of life, and significantly extend the survival of dogs affected with this previously hopeless disease.

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