MEGANUCLEASE VARIANTS CLEAVING A DNA TARGET SEQUENCE FROM THE HUMAN LYSOSOMAL ACID ALPHA-GLUCOSIDASE GENE AND USES THEREOF

摘要

An l-Crel variant, wherein one of the two l-Crel monomers has at least two substitutions, one in each of the two functional subdomains of the LAGLIDADG core domain situated respectively from positions 26 to 40 and 44 to 77 of l-Crel, said variant being able to cleave a DNA target sequence from the human lysosomal acid α-glucosidase gene. Use of said variant and derived products for the prevention and the treatment of pathological conditions caused by a mutation in the human lysosomal acid α-glucosidase gene (Pompe's disease).