Max-delbruck-centrum Fur Molekulare Medizin

University of Minnesota

Max-delbruck-centrum Fur Molekulare Medizin in Der Helmholtz-gemeinschaft

Genetic Information Research Institute

Max-delbruck-centrum Fur Molekulare Medizin (mdc)

Julius-maximilians-universitat Wurzburg

T-curx Gmbh

Zoltan Ivics

Carsten Rudolph

Zsuzsanna Izsvak

Perry B Hackett

Namitha Manoj

Carmen-Anisia Judis

Andrea Katzer

Johannes Geiger

Lajos Mates

Karl J Clark

Tilmann Buerckstuemmer

Vladimir Vyacheslavovich Kapitonov

Michael Hudecek

Ivana Grabundzija

Csaba Miskey

Scott C Fahrenkrug

Hatem Zayed

Manish Aneja

Marta Holstein

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

The invention relates to a system and method for introducing DNA into cells. In particular, the invention relates to a method for introducing single or multiple copies of a DNA sequence or gene of interest into a cell comprising providing: a) a 'copy and paste' transposase; and b) a construct comprising a DNA sequence or gene of interest flanked by a 'copy and paste' transposon terminal sequence, such as an LTS or RTS. A novel &#x201c;copy and paste&#x201d; transposon of the Helitron family is described along with systems for using the corresponding transposase in methods for introducing DNA into cells, for example, to generate cell lines for use in protein production, cell and gene therapy or as reference standards.

Replicative transposon system

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 refers to a gene transfer system for stably introducing nucleic acid(s) into the DNA of a cell by using a member of the human mariner transposases. The invention further refers to this transposase and to transposons used in the inventive gene transfer system, comprising a nucleic acid sequence with flanking repeats (IRs and/or IR/DRs). Furthermore, applications of this 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.

Reconstructed human mariner transposon capable of stable gene transfer into chromosomes in vertebrates

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

Zoltan Ivics