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US 2014/0193906 A1Published application
Screening methods for spinal muscular atrophy
AssigneeIndiana University Research and Technology Corporation
InventorsElliot Androphy, Gregory D. Cuny, Jonathan Cherry, Marcie A. Glicksman
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Abstract
Disclosed herein are compositions and methods for treatment of spinal muscular atrophy (SMA). In certain embodiments, compounds are provided that increase full-length survival of motor neuron (SMN) protein production by an SMN2 gene.
Claims19
A vector comprising:
- a survival motor neuron (SMN) promoter selected from the group consisting of a survival motor neuron 1 (SMN1) promoter and a survival motor neuron 2 (SMN2) promoter;
- a transcription start site;
- a nucleic acid encoding exons 1-8 of the SMN gene and encoding introns 6-8 of the SMN gene;
- and a reporter gene.
1 more element
A vector comprising:
- a survival motor neuron 1 (SMN1) promoter;
- a transcription start site;
- a nucleic acid encoding exons 1-8 of the SMN1 gene and encoding introns 6-8 of the SMN1 gene;
- and a reporter gene.
1 more element
A vector comprising:
- a survival motor neuron 2 (SMN2) promoter and a survival motor neuron 2 (SMN2) promoter;
- a transcription start site;
- a nucleic acid encoding exons 1-8 of the SMN2 gene and encoding introns 6-8 of the SMN2 gene;
- and a reporter gene.
1 more element
Dependencies are inferred from the claim text.