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    • rAAV-based gene therapy for molybdenum cofactor deficiency type B 

      Jakubiczka-Smorag, Joanna (2015-06-29)
      Molybdenum cofactor (MoCo) deficiency type A is thus far only one curable type of MoCo deficiency. In the present study, a mouse model for MoCo deficiency type B (Mocs2-/-) was generated, characterised and used for preclinical ...