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Company Dimension Therapeutics Inc.
Description Adeno-associated viral (AAV) vector delivering argininosuccinate synthase 1 (ASS; ASS1)
Molecular Target Argininosuccinate synthase 1 (ASS) (ASS1)
Mechanism of Action 
Therapeutic ModalityGene therapy: Viral vector: Adeno-associated virus (AAV)
Latest Stage of DevelopmentDiscovery
Standard IndicationUrea cycle disorder (UCD)
Indication DetailsTreat citrullinemia type I
Regulatory Designation

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