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.
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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