FIELD: biotechnology; medicine.
SUBSTANCE: invention relates to biotechnology and medicine. The present invention relates to a modified FXN gene providing increased expression of the encoded frataxin protein, which can be used to treat Friedreich's ataxia.
EFFECT: modified FXN gene provides increased expression of the encoded frataxin protein, which can be used to treat Friedreich's ataxia.
38 cl, 6 dwg, 8 tbl, 3 ex
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