Meganuclease variants cleaving a dna target sequence from...

Details Meganuclease variants cleaving a dna target sequence from... Meganuclease variants cleaving a dna target sequence from...

C - Chemistry, Metallurgy

07

K

C07K 14/435 (2006.01) A01K 67/00 (2006.01) A61K 38/46 (2006.01) C12N 9/22 (2006.01) C12N 15/90 (2006.01)

Patent

CA 2695495

An I-CreI variant, wherein at least one of the two I-CreI 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 I--CreI, said variant being able to cleave a DNA target sequence from the human IL2RG gene. Use of said variant and derived prod-ucts for the prevention and the treatment of X-linked severe combined immunodeficiency.

L'invention porte sur un variant de I-CreI, dans lequel au moins l'un des deux monomères I-CreI a au moins 2 substitutions, une dans chacun des deux sous-domaines fonctionnels du domaine de noyau LAGLIDADG situés respectivement aux positions 26 à 40 et 44 à 77 de I-CreI, ledit variant étant apte à cliver une séquence cible d'ADN provenant du gène IL2RG humain. L'invention porte également sur l'utilisation dudit variant et sur des produits dérivés pour la prévention et le traitement d'une immunodéficience combinée grave liée à X.

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