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Safety and Liver Transduction Efficacy of rAAV5- cohPBGD in Nonhuman Primates: A Potential Therapy for Acute Intermittent Porphyria

26 Sept 2013

Abstract excerpt

Acute intermittent porphyria (AIP) results from haplo-insufficient activity of porphobilinogen deaminase (PBGD) and is characterized clinically by life-threatening, acute neurovisceral attacks. To date, liver transplantation is the only curative option for AIP. The aim of the present preclinical nonhuman primate study was to determine the safety and transduction efficacy of an adeno-associated viral vector...

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PMID: 24070415
DOI: 10.1089/hum.2013.166

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