Patent ID: 8986935
Filing Date: 2015-03-24
Classification: A61K,A61P,C12N,C12Q

Abstract:
1. A method for the identification of a compound that produces a stabilized SMNΔEx7 protein comprising: (A) contacting a compound with either a host cell containing an mRNA transcript transcribed from a nucleic acid construct, or a composition comprising a cell-free extract and an mRNA transcript transcribed from a nucleic acid construct, wherein the nucleic acid construct comprises, in 5′ to 3′ order: (B) detecting the activity or amount of a fusion protein translated from the mRNA transcript, wherein an increase in the activity or amount of the fusion protein translated from the mRNA transcript in the presence of a compound when compared to: indicates that the compound produces a stabilized SMNΔEx7 protein; and (C) contacting a compound that increases the activity or amount of the fusion protein translated from the mRNA transcript with a cell that produces SMNΔEx7 protein and assaying the ability of the compound to increase the level of stabilized SMNΔEx7 protein in the cell in the presence of the compound as compared to the level of SMNΔEx7 protein in the cell in the presence of the compound, wherein an increase in the level of stabilized SMNΔEx7 protein relative to the level of SMNΔEx7 protein indicates that a compound that produces a stabilized SMNΔEx7 protein is identified, wherein said stabilized SMNΔEx7 protein comprises the amino acid sequence of SEQ ID NO:2, SEQ ID NO:3, SEQ ID NO:4, or SEQ ID NO:5.