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

12

N

C12N 9/22 (2006.01)

Patent

CA 2669313

An I-CreI variant or a single-chain derivative having at least one substitution in one of the two functional subdomains of the LAGLIDADG core domain situated from positions 26 to 40 and 44 to 77 of I-CreI, and being able to cleave a DNA target sequence from the HPRT gene having a nucleotide sequence of SEQ ID NO: 1 to 14. Use of said variant for inducing a site-specific modification in the HPRT gene, for therapeutic (gene therapy of Lesch-Nyhan syndrome) or non-therapeutic purpose (engineering of transgenic animals and recombinant cell lines).

Variant I-Crel ou dérivé à chaîne unique présentant au moins une substitution dans l'un des deux sous-domaines fonctionnels du domaine principal LAGLIDADG compris entre les positions 26 à 40 et 44 à 77 de I-Crel et qui est capable de cliver une séquence cible d'ADN à partir du gène HPRT ayant une séquence nucléotidique de SEQ ID: 1 à 14. Utilisation dudit variant pour induire une modification spécifique du site dans le gène HPRT à des fins thérapeutiques (thérapie génique pour le syndrome de Lesch-Nyhan) ou non thérapeutiques (obtention par génie génétique d'animaux transgéniques et de lignes de cellules de recombinaison).

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