C - Chemistry – Metallurgy – 12 – N
Patent
C - Chemistry, Metallurgy
12
N
C12N 9/16 (2006.01) A61K 48/00 (2006.01) C12N 15/09 (2006.01) C12N 15/55 (2006.01) C12N 15/63 (2006.01) C12N 15/82 (2006.01) C12N 15/85 (2006.01) G01N 33/48 (2006.01) A01K 67/027 (2006.01) C12N 5/10 (2006.01)
Patent
CA 2657767
An I-CreI variant, wherein one of the 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 a RAG gene. Use of said variant and derived products for the prevention and the treatment of a SCID syndrome associated with a mutation in a RAG gene.
La présente invention concerne un variant I-CreI dont l'un des monomères I-CreI comporte au moins deux substitutions, une dans chacun des deux sous-domaines fonctionnels du domaine central LAGLIDADG allant respectivement des positions 26 à 40 et 44 à 77 de I-CreI, ledit variant étant capable de cliver une séquence d'ADN cible d'un gène RAG. L'invention porte également sur l'utilisation dudit variant et de produits dérivés pour la prévention et le traitement d'un syndrome d'immunodéficience combinée grave (SCID) associé à une mutation d'un gène RAG.
Arnould Sylvain
Grizot Sylvestre
Cellectis
Robic
LandOfFree
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