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MODIFIED FRIEDREICH ATAXIA GENES AND VECTORS FOR GENE THERAPY.
MODIFIED FRIEDREICH ATAXIA GENES AND VECTORS FOR GENE THERAPY.
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机译:改良的弗里德里希·阿塔克西亚基因和基因治疗载体。
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摘要
The present invention relates to a modified FXN gene providing for increased expression of the encoded protein frataxin that can be used for treatment of Friedreich ataxia.
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