02210: Gene Therapy for Canine Degenerative Myelopathy
Grant Status: Open
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. 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), a neurodegenerative disorder very similar to canine DM. It is also shown that reduction of mutant SOD1 in ALS mouse models provides beneficial effects. Hence, therapeutic approaches to reduce the expression of mutant SOD1 in DM-affected dogs may improve survival and preserve neurologic function. 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. 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.
None at this time.
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