Synthetic mammalian chromosome and methods for construction

Details Synthetic mammalian chromosome and methods for construction Synthetic mammalian chromosome and methods for construction

C - Chemistry, Metallurgy

12

N

C12N 15/11 (2006.01) A61K 31/70 (2006.01) A61K 48/00 (2006.01) C12N 15/10 (2006.01) C12N 15/85 (2006.01) C12N 15/90 (2006.01)

Patent

CA 2224114

The invention relates to the field of gene therapy, gene expression, and vectors for these uses. In particular, the invention relates to a method for producing structurally intact large repeating units of DNA, especially useful for the stable cloning of alpha satellite DNA, and to the development and use of a synthetic or artificial chromosome for gene expression and gene therapy in mammals, and especially humans. The invention allows the controlled construction of stable synthetic or artificial chromosomes constructed from isolated segments of purified DNA. Functional minimal segments preferably include centromeric DNA, telomeric DNA, and genomic DNA. The artificial chromosome performs the essential chromosomal functions of naturally-occurring chromosomes so as to permit the chromosome to function as an effective vector for gene therapy.

L'invention concerne le domaine de la thérapie et de l'expression géniques, ainsi que des vecteurs destinés à ces utilisations. L'invention se rapporte notamment à un procédé de production de grandes unités de répétition, structurellement intactes, d'ADN, spécialement utiles pour cloner de manière stable l'ADN satellite alpha, ainsi qu'au développement et à l'utilisation d'un chromosome synthétique ou artificiel destiné à l'expression et à la thérapie géniques chez les mammifères et notamment chez l'homme. L'invention permet la construction régulée de chromosomes stables, synthétiques ou artificiels, construits à partir de segments isolés d'ADN purifié. Des segments fonctionnels minimaux comprennent, de préférence, de l'ADN centromère, télomère et génomique. Ce chromosome artificiel remplit les fonctions chromosomiques essentielles de chromosomes naturels, de manière à pouvoir fonctionner en tant que vecteur efficace destiné à la thérapie génique.

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