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HIGHLY SPECIFIC NUCLEASES FOR GENE TARGETING AND GENE THERAPY Ref-No: TA-TM631 BACKGROUND The new properties eliminate known disadvantages of conven-tionally used chimeric nucleases and allow to specifically modify genomes e.g. for biotechnological or gene therapeutic applications. The high specificity and preference of the new chimeric nucleases is based on a fusion of TALE proteins as DNA binding modules with a highly specific restriction endonuclease, PvuII, as DNA cleavage module. TransMIT Gesellschaft für Technologietransfer mbH Niklas Günther +49 (641) 94364-53 [email protected] www.transmit.de PROBLEM Chimeric nucleases known at present share the disadvantage that these enzymes cleave DNA in the genome also unspecifically, a feature resulting in unacceptable cytotoxicity. DEVELOPMENT STATUS SOLUTION PATENT SITUATION Within the scope of gene targeting it is therefore now possible to introduce desired genes or genome segments into cleavage sites with high specificity. EP granted DE pending GB pending Proof of concept CATEGORIES //Therapy and therapeutics //Red biotechnology //Molecular biology, genetics www.inventionstore.de 1/2 Powered by TCPDF (www.tcpdf.org) ADVANTAGES In comparison with chimeric nucleases commercially available at present, the new fusion proteins offer significant advantages: 1. They have a strong preference for unique DNA cleavage sites. 2. They cleave genomic DNA with high specificity, while unspecific (offtarget) DNA-cleavage is prevented. 3. The application of the new chimeric nucleases results in a reduced toxicity (off-target effect). SCOPE OF APPLICATION The invention is of interest for companies who develop and manufacture products in the fields of biotechnology and/or gene therapy. www.inventionstore.de 2/2