ALPHA-SARCOGLYCAN GENE TRANSFER INCREASE USING MODIFIED ITR SEQUENCES

The present invention relates to a gene expression construct comprising an expression cassette containing a transgene encoding an alpha-sarcoglycan protein and modified ITR sequences, said transgene encoding the alpha-sarcoglycan protein consisting of the sequence of SEQ ID NO: 3 or a functional variant thereof having a polynucleotide sequence that is at least 80% identical to SEQ ID NO: 3; said modified ITR sequences consisting of - a modified 5'-ITR sequence of SEQ ID NO: 1; and - a modified 3'-ITR sequence of SEQ ID NO: 2. The invention also relates to a recombinant vector comprising the gene expression construct of the invention, said gene expression construct or recombinant vector can be included in a gene therapy and used as a medicament, in particular for the treatment of limb girdle muscular dystrophy by sarcoglycanopathy.