Download highly specific nucleases for gene targeting and

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
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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.
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