University of Minnesota

Max-Delbruck-Centrum Fur Molekulare Medizin

Max-Delbruck-Centrum Fur Molekulare Medizin (Mdc)

Zsuzsanna Izsvak

Zoltan Ivics

Perry B Hackett

Namitha Manoj

Carmen-Anisia Judis

Andrea Katzer

Lajos Mates

Karl J Clark

Scott C Fahrenkrug

Zoltán Ivics

Hatem Zayed

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 transposon, 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.

Transposon, gene transfer system and method of using the same

Nucleic acids encoding SB10 variants

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.

Polypeptide variants of sleeping beauty transposase

The present invention relates to a targeting system comprising, preferably as distinct components (a) a transposon which is devoid of polynucleotide encoding a functional transposase comprising (aa) a polynucleotide of interest and; (ab) a DNA sequence specifically recognized by a DNA binding domain; and (ba) a fusion protein comprising (i) said DNA binding domain; or (ii) a (poly)peptide binding domain binding to a (poly)peptide comprising said DNA binding domain; and (iii) a DNA targeting domain; or (iv) a (poly)peptide binding domain that binds to a cellular or engineered (poly)peptide that comprises a DNA targeting domain; or (bb) a polynucleotide encoding the fusion protein of (ba); and (ca) a transposase or a fragment or derivative thereof having transposase function; or (cb) a polynucleotide encoding the transposase or fragment or derivative thereof having transposase function of (ca).

Transposon-based targeting system

The present invention provides for transposon vectors encoding expression control region-traps and gene-traps. Also provided are dicistronic vectors. Certain embodiments of the invention contain internal ribosome entry sites.

Nucleic acid transfer vector for the introduction of nucleic acid into the DNA of a cell

This invention relates to a system for introducing nucleic acid into the DNA of a cell. The system includes the use of a member of the SB family of transposases (SB) or nucleic acid encoding the transposase and a nucleic acid fragment that includes a nucleic acid sequence with flanking inverted repeats. The transposase recognizes at least a portion of an inverted repeats and incorporates the nucleic acid sequence into the DNA. Methods for use of this system are discussed.

DNA-based transposon system for the introduction of nucleic acid into DNA of a cell

Growing community of inventors

Berlin, Germany

Zsuzsanna Izsvak