MODIFIED HUMAN U1SNRNA MOLECULE, A GENE ENCODING FOR THE MODIFIED HUMAN U1SNRNA MOLECULE, AN EXPRESSION VECTOR INCLUDING THE GENE, AND THE USE THEREOF IN GENE THERAPY OF FAMILIAL DYSAUTONOMIA AND SPINAL MUSCULAR ATROPHY
The invention provides a modified human U1snRNA molecule, capable of correcting the skipping of an exon caused by a mutation localized in the sequence comprised between 50 base pairs upstream and 20 base pairs downstream of an exon, wherein a portion of a single-stranded nucleotide sequence of the 5′ region of the wild-type human U1snRNA is replaced by a single-stranded binding nucleotide sequence, wherein the binding nucleotide sequence is selected from the group consisting of: uggcgcuua, aauggcgcu, aguacaauggcgc (SEQ ID NO: 87), gcaaacaguacaau (SEQ ID NO: 88), ucgcaaacaguaca (SEQ ID NO: 89), gcaaacagu, cuagucgcaaac (SEQ ID NO: 90), uacaaaaguaagauuca (SEQ ID NO: 83), aaaccauaaaguuuuacaa (SEQ ID NO: 84) and caaaccauaaaguuuua (SEQ ID NO: 96).
