Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPL S447X) gene therapy for lipoprotein lipase deficiency: An open-label trial

Gene Therapy

Volumn 20, Issue 4, 2013, Pages 361-369

  Gaudet, D ^a,b   Methot J ^a,b   Dery S ^a   Brisson, D ^a,b   Essiembre, C ^a   Tremblay, G ^a   Tremblay, K ^a,b   De Wal, J ^c   Twisk, J ^c   Van Den Bulk, N ^c   Sier Ferreira, V ^c   Van Deventer, S ^c  

^a CHICOUTIMI HOSPITAL   (Canada)

^b UNIVERSITÉ DE MONTRÉAL   (Canada)

^c Amsterdam Molecular Therapeutics (AMT) BV   (Netherlands)

Author keywords

AAV1 LPL S447X; alipogene tiparvovec; familial chylomicronemia; LPL deficiency

Indexed keywords

ALIPOGENE TIPARVOVEC; CAPSID PROTEIN; CREATINE KINASE; CYCLOSPORIN A; LACTATE DEHYDROGENASE; LIPOPROTEIN LIPASE; MYCOPHENOLIC ACID 2 MORPHOLINOETHYL ESTER; TRIACYLGLYCEROL;

ACUTE PANCREATITIS; ADULT; ARTICLE; CLINICAL ARTICLE; CREATINE KINASE BLOOD LEVEL; DIET RESTRICTION; DRUG DOSE ESCALATION; DRUG EFFICACY; DRUG SAFETY; FEMALE; FOLLOW UP; GASTROCNEMIUS MUSCLE; GENE EXPRESSION; GENE THERAPY; GENETIC VARIABILITY; HUMAN; HYPERLIPOPROTEINEMIA TYPE 1; IMMUNE RESPONSE; IMMUNOGENICITY; IMMUNOSUPPRESSIVE TREATMENT; INJECTION SITE CONTUSION; INJECTION SITE EDEMA; INJECTION SITE PAIN; INTERVENTION STUDY; LACTATE DEHYDROGENASE BLOOD LEVEL; LOW FAT DIET; MALE; MUSCLE BIOPSY; OPEN STUDY; PANCREATITIS; PHYSICAL EXAMINATION; PRIORITY JOURNAL; SEROTYPE; SIDE EFFECT; SPINAL ANESTHESIA; TRANSGENE; TREATMENT DURATION; TRIACYLGLYCEROL BLOOD LEVEL; VASTUS LATERALIS MUSCLE; VASTUS MEDIALIS MUSCLE;

ADULT; DEPENDOVIRUS; DRUG TOLERANCE; FASTING; FEMALE; GENETIC THERAPY; HUMANS; HYPERLIPOPROTEINEMIA TYPE I; IMMUNOSUPPRESSIVE AGENTS; LIPOPROTEIN LIPASE; MALE; MIDDLE AGED; PANCREATITIS; PROSPECTIVE STUDIES; TREATMENT OUTCOME; TRIGLYCERIDES;

EID: 84875962928     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/gt.2012.43     Document Type: Article

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