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Technology Validation

Tremendous scientific and technical progress has been made towards the ultimate goal of correcting inborn errors in genes. One of the most promising fields amid all this progress lies in oligonucleotide technology. The Nobel Prize in Physiology or Medicine in 2006, awarded for RNA interference (RNAi), has validated many techniques for oligonucleotide-based therapies. Furthermore, in 2007, the Nobel Prize was awarded for gene targeting, which encompasses the underlying science for transferring genes into the genome. TGA takes advantage of the techniques and science inherent in these discoveries in order to target and correct errors in the gene itself.

The following studies exhibit third-party validation of the success of OrphageniX technology in animal models of important diseases:

TGA corrects mouse retina gene in an in vivo model for retinitis pigmentosa, increases survival of photoreceptors. Experiment includes repeated and confirmed results. AAndrieu-Soler, C., Halhal, M., Boatright, J.H., Padove, S.A., Nickerson, J.M., Stodulkova, E., Stewart, R.E., Ciavatta, V.T., Doat, M., Jeanny, J.C., de Bizemont, T., Sennlaub, F., Courtois, Y., Behar-Cohen, F. (2007) Single-stranded oligonucleotide-mediated in vivo gene repair in the rd1 retina. Molecular Vision. 13, 692-706

TGA corrects dystrophin gene mutation in mouse model of muscular dystrophy
Bertoni, C., Morris, G.E., and Rando, T.A. (2005) Strand bias in oligonucleotide-mediated dystrophin gene-editing. Human Molecular Genetics. 14:2, 221-233.

TGA corrects mutation in TTR gene to halt production of harmful transthretin protein in liver cells Nakamura, M., Ando, Y., Nagahara S., Sano, A., Ochiya, T., Maeda, S., Kawaji, T., Ogawa, M., Hirata, A., Terazaki, H., Haraoka, K., Tanihara, H., Ueda, M., Uchino, M., and Yamamura K. (2004) Targeted conversion of the transthyretin gene in vitro and in vivo. Gene Therapy. 11, 838-846.

TGA corrects mutation and restores enzymatic activity in skin fibroblast cells for Pompe Disease Lu, I.L, Lin, C.Y., Lin, S.B., Chen, S.T., Yeh, L.Y., Yang, F.Y., and Au, L.C. (2003) Correction/mutation of acid -D-glucosidase gene by modified single-stranded oligonucleotides: in vitro and in vivo studies. Gene Therapy. 10, 1910-1916