Gene editing system corrects disease mutation in cells

Mutations cause a form of Beta-thalassemia

LEXINGTON, Ky. (Aug. 8, 2014) — 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.

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.

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.