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Elliot Androphy

UMassMed Faculty Page



  • This technology provides vectors that increase survival of motor neuron (SMN) protein production by an SMN2 gene and methods for treatment of spinal muscular atrophy (SMA). Further discloses is a clonal second generation SMN-luciferase reporter cell line that combines the strengths of both the promoter-based assay and a previous splicing reporter. This assay is much more robust, has lower well-to-well variation, and displays more stable luciferase expression that does not change with serial passage that can be used to identify potential therapeutic agents for SMA.

Title: Arylthiazolyl Piperidines as Modulators of Survival Motor Neuron (SMN) Production. UMMS10-63; Patent Pending. 

  • This invention discloses aryl substituted thiazol-2-yl-piperidines and other related compounds that modulate production of the protein, Survival Motor Neuron (SMN), that plays a role in the treatment of spinal muscular atrophy. These newly identified compounds can modulate SMN2 protein levels and ultimately increase motor neuron survival. Pharmacological composition and methods for treating spinal muscular atrophy are further shown.


Innovation TopicsBiologicsResearch ToolsNeurologySpinal muscular atrophySmall Molecules