Transposagen's Footprint-Free(TM) Gene Editing System Corrects Beta-Thalassemia Disease Mutation in Human iPS Cells

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| Source: Transposagen Biopharmaceuticals, Inc.
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How Footprint-Free(TM) Gene Editing Works
  • 1- Start with NextGEN(TM) CRISPR and/or XTN(TM) TALENs to stimulate homologous recombination at the target site.
  • 2- Use a Footprint-Free(TM) Gene Editing donor plasmid from our Multivector(tm) portfolio for targeted integration and post-selection enrichment of the desired gene edit.
  • 3- Following selection, use Excision-Only piggyBac(TM) (PBx) to remove selectable markers, leaving behind a scarless and correctly edited genomic DNA.

LEXINGTON, Ky., Aug. 8, 2014 (GLOBE NEWSWIRE) -- In the latest demonstration of Transposagen's Footprint-Free™ Gene Editing System, published this week in Genome Research, scientists from the University of California San Francisco corrected mutations in the HBB gene, which cause a form of Beta-thalassemia. Transposagen commercializes the Footprint-Free™ Gene Editing System, which combines the industry's most precise, efficient, and flexible site-specific nucleases, NextGEN™ CRISPRs and XTN™ TALENs, with its exclusive piggyBac™ transposon system, resulting in the only commercially-available method capable of seamless excision of resistance or reporter genes. Although Transposagen scientists were not involved in the Beta-thalassemia work, Transposagen also offers custom engineering of iPS and other cell lines on a fee-for-service basis.

A photo accompanying this release is available at http://www.globenewswire.com/newsroom/prs/?pkgid=26985.

By utilizing the Footprint-Free™ system researchers have the ability to efficiently and precisely edit as little as a single nucleotide in any genome without leaving any undesired mutations and with the ability to select for rare events. The technology can be used to correct any disease-causing mutation in cells that have been removed from a patient. The corrected cells may eventually be put back in the patient to cure or mitigate the disease. The system may also be utilized in research and development applications, such as for producing disease-specific cell lines or isogenic control cell lines.

Footprint-Free™ Gene Editing

Transposagen's Footprint-Free™ Gene Editing System, was recently utilized by researchers from University of California-San Francisco to seamlessly correct mutations in the HBB gene, which cause a form of Beta-thalassemia in iPS cells. Transposagen commercializes the technology which can be used to correct any disease-causing mutation in cells that have been removed from a patient. The corrected cells may eventually be put back in the patient to cure or mitigate the disease. The system may also be utilized in research and development applications, such as for producing disease-specific cell lines or isogenic control cell lines.

Jack Crawford
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