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TALE-Frataxin Gene Therapy
AmorChem Canada flag Canada
Abstract ID:
TALE-Frataxins have been designed and tested to augment endogenous Frataxin levels in mouse cells as well as in human fibroblasts derived from Friedreich\'s Ataxia patients. Further optimization in animal modeling of the disease will be done....
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Friedreich's Ataxia (FA) is an autosomal recessive neurodegenerative and cardiac disease due to a trinucleotide (GAA) repeat expansion in intron 1 of the FXN (FXN) gene. This mutation does not modify the amino acids of the FXN protein but does reduce its expression. Thus FA is representative of a typical haplo-insufficiency disease.

The level of FXN is related to the number of the trinucleotide repeats, it is 30% in patients with 200 GAA repeats and only 5% in patients with 900 GAA repeats. However, carriers of this disease produce about only 50% of the normal level of FXN but do not develop symptoms.

Type of Business Relationship Sought
Out-licensing or collaboration
Last Updated Jun 2016
Technology Type THERAPEUTIC
Phase of Development PRECLINICAL