Neocentromere-based mini-chromosomes or artificial chromosomes

Details Neocentromere-based mini-chromosomes or artificial chromosomes Neocentromere-based mini-chromosomes or artificial chromosomes

C - Chemistry, Metallurgy

12

N

C12N 15/64 (2006.01) C12N 15/68 (2006.01) C12N 15/79 (2006.01) C12N 15/81 (2006.01) C12N 15/82 (2006.01) C12N 15/85 (2006.01) A61K 48/00 (2006.01)

Patent

CA 2431884

The present invention is directed generally to a defined or isolated nucleic acid molecule encompassing a neocentreomere or a functional derivative thereof or a latent, synthetic or hybrid form thereof and its use inter alia in developing a range of eukaryotic mini-chromosomes and artificial chromosomes including mammalian (e.g. human) and non-mammalian mini-chromosomes and artificial chromosomes. The present invention further provides a telomere- associated chromosome truncation (TACT) approach to develop mini-chromosomes, but is not limited to this approach. It is directed to any truncation approach that produces a neocentromere-containing mini-chromosome, or any transfection approach using cloned or pre-fabricated DNA that provides neocentromere function in the construction of artificial chromosome. Such mini-chromosomes and artificial chromosomes are useful in a range of genetic therapies.

De manière générale, l'invention concerne la définition ou l'isolation d'une molécule d'acides nucléiques englobant un néocentromère ou un dérivé fonctionnel de celui-ci, ou une forme hybride ou synthétique, latente, de celui-ci; elle concerne également l'utilisation de cette molécule, entre autres, pour mettre au point une gamme de mini-chromosomes et chromosomes artificiels eucaryotes, notamment des mini-chromosomes et chromosomes artificiels, mammifères et non mammifères. L'invention concerne en outre, sans y être limitée, une approche de troncature de chromosome associé à un télomère, servant à mettre au point des mini-chromosomes, et elle porte notamment sur toute approche de troncature produisant un mini-chromosome contenant un néocentromère, ou toute approche de transfection mettant en oeuvre un ADN cloné ou préfabriqué, lequel apporte la fonction de néocentromère dans la construction du chromosome artificiel. De tels mini-chromosomes et chromosomes artificiels sont utiles dans un éventail de thérapies géniques.

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