MEGANUCLEASE VARIANTS CLEAVING A DNA TARGET SEQUENCE FROM THE BETA-2-MICROGLOBULIN GENE AND USES THEREOF

摘要

An I-CreI variant which has at least two substitutions, one in each of the two functional subdomains of the LAGLIDADG core domain situated from positions 26 to 40 and 44 to 77 of I-Crel, said variant being able to cleave a DNA target sequence from the beta-2 microglobulin gene. Use of said variant and derived products for the prevention and the treatment of xenograft rejection and pathological conditions associated with a fibrillar conformation of the beta-2 microglobulin,, as well as for the engineering of transgenic animals and recombinant cell lines expressing an heterologous protein of interest.