Patent ID: 8633019
Filing Date: 2014-01-21
Classification: A61P,C07D,C07K,C12N,C12Q

Abstract:
1. A nucleic acid construct comprising a minigene which comprises, in 5′ to 3′ order: a start codon, the nucleic acid residues of exon 6 of human survival motor neuron (SMN), the nucleic acid residues of intron 6 of human SMN, the nucleic acid residues of exon 7 of human SMN2, the nucleic acid residues of intron 7 of human SMN, a fragment of exon 8 of human SMN, and the nucleic acid residues of the coding sequence of a reporter gene lacking a start codon, wherein a single adenine residue is inserted after nucleic acid residue 48 and before nucleic acid residue 49 of the nucleic acid residues of exon 7 of human SMN2, and wherein the first codon of the coding sequence of the reporter gene and the start codon of the minigene are in the same open reading frame.