Meganuclease variants cleaving a dna target sequence from...

C - Chemistry – Metallurgy – 12 – N

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C12N 9/22 (2006.01) A61K 38/16 (2006.01) A61K 48/00 (2006.01) A61P 37/06 (2006.01) C12N 15/55 (2006.01) C12N 15/90 (2006.01)

Patent

CA 2678709

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.

Variant I-Crel ayant au moins deux substitutions, chacune dans un des deux sous-domaines fonctionnels du domaine principal LAGLIDADG situé entre les positions 26 et 40 et 44 et 77 de I-Crel, ledit variant étant capable de cliver une séquence d'ADN cible du gène de la bêta-2-microglobuline. Utilisation de ce variant et des ses produits dérivés pour la prévention et le traitement du rejet de xénogreffons et des conditions pathologiques associées à la conformation de la bêta-2-microglobuline., ainsi que pour l'obtention d'animaux transgéniques et de lignées cellulaires de recombinaison exprimant une protéine hétérologue d'intérêt.

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