Hyperactive variants of the transposase protein of the...

C - Chemistry – Metallurgy – 12 – N

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C12N 9/22 (2006.01) C12N 15/90 (2006.01)

Patent

CA 2692501

The present invention refers to hyperactive variants of a transposase of the transposon system Sleeping Beauty (SB). The invention further refers to corresponding nucleic acids producing these variants, to a gene transfer system for stably introducing nucleic acid(s) into the DNA of a cell by using these hyperactive variants of a transposase of the transposon system Sleeping Beauty (SB) and to transposons used in the inventive gene transfer system, comprising a nucleic acid sequence with flanking repeats (IRs and/or RSDs). Furthermore, applications of these transposase variants, the transpsoson, or the gene transfer system are also disclosed such as gene therapy, insertional mutagenesis, gene discovery (including genome mapping), mobilization of genes, library screening, or functional analysis of genomes in vivo and in vitro. Finally, pharmaceutical compositions and kits are also encompassed.

La présente invention concerne des variantes hyperactives d'une transposase du système de transposon Sleeping Beauty (SB). L'invention concerne également les acides nucléiques correspondants qui produisent ces variantes, un système de transfert de gènes pour l'introduction stable d'acide(s) nucléique(s) dans l'ADN d'une cellule au moyen de ces variantes hyperactives d'une transposase du système de transposon Sleeping Beauty (SB) et des transposons utilisés dans le système de transfert de gènes selon l'invention, qui comprennent une séquence d'acides nucléiques avec des répétitions flanquantes (IR et/ou RSD). L'invention concerne également des applications de ces variantes de transposase, le transposon ou le système de transfert de gènes, ainsi qu'une thérapie génique, une mutagénèse insertionnelle, la découverte de gènes (y compris le mappage du génome), la mobilisation de gènes, le criblage de bibliothèque ou l'analyse fonctionnelle de génomes in vivo et in vitro. L'invention concerne également des compositions pharmaceutiques et des trousses.

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