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COMPOSITIONS AND METHODS FOR TREATMENT OF TYPE VII COLLAGEN DEFICIENCIES
COMPOSITIONS AND METHODS FOR TREATMENT OF TYPE VII COLLAGEN DEFICIENCIES
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机译:治疗VII型胶原蛋白缺乏症的组合物和方法
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摘要
The present invention relates to self-inactivating lentiviral vectors comprising the COL7A1 gene or a functional variant thereof and its use in a method for the treatment of Type VII collagen deficiency, such as dominant dystrophic epidermolysis and recessive dystrophic epidermolysis.
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