Gene therapy in combination with nitrogen scavenger pretreatment corrects biochemical and behavioral abnormalities of infant citrullinemia type 1 mice

HIGHLIGHTS

• who: Andrea Bazo and colleagues from the Division of Gene Therapy and Regulation of Gene Expression, CIMA, University of Navarra, Pamplona, Spain have published the paper: Gene Therapy in Combination with Nitrogen Scavenger Pretreatment Corrects Biochemical and Behavioral Abnormalities of Infant Citrullinemia Type 1 Mice, in the Journal: (JOURNAL)
• what: This study shows the potential of VTX-804 gene therapy for future clinical translation to CTLN1 patients. The authors assessed the therapeutic effect of a recombinant AAVAnc80 vector expressing human ASS after its administration to 3-week-old Ass1fold mice treated from birth to weaning . . .
  

   

  

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