(Q34311144)

1 August 2017

title

Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial (English)

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1 August 2017

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D Gaudet

1

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1 August 2017

J Méthot

2

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1 August 2017

S Déry

3

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1 August 2017

D Brisson

4

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1 August 2017

C Essiembre

5

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1 August 2017

G Tremblay

6

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1 August 2017

K Tremblay

7

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1 August 2017

J de Wal

8

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1 August 2017

J Twisk

9

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1 August 2017

N van den Bulk

10

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1 August 2017

V Sier-Ferreira

11

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1 August 2017

S van Deventer

12

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1 August 2017

language of work or name

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21 June 2012

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1 August 2017

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1 August 2017

volume

20

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1 August 2017

issue

4

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1 August 2017

page(s)

361-369

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Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing α1-antitrypsin: interim results

1 August 2017

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Review of the clinical development of alipogene tiparvovec gene therapy for lipoprotein lipase deficiency

6 July 2018

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Effects of transient immunosuppression on adenoassociated, virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy

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Lipoprotein lipase S447X: a naturally occurring gain-of-function mutation

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Enhanced susceptibility to pancreatitis in severe hypertriglyceridaemic lipoprotein lipase-deficient mice and agonist-like function of pancreatic lipase in pancreatic cells.

6 July 2018

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Gene therapy for lipoprotein lipase deficiency: working toward clinical application

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Correction of feline lipoprotein lipase deficiency with adeno-associated virus serotype 1-mediated gene transfer of the lipoprotein lipase S447X beneficial mutation

21 January 2018

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Long-term correction of murine lipoprotein lipase deficiency with AAV1-mediated gene transfer of the naturally occurring LPL(S447X) beneficial mutation

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Intramuscular administration of AAV1-lipoprotein lipase S447X lowers triglycerides in lipoprotein lipase-deficient patients

21 January 2018

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Evidence of multiyear factor IX expression by AAV-mediated gene transfer to skeletal muscle in an individual with severe hemophilia B.

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Alipogene tiparvovec, an adeno-associated virus encoding the Ser(447)X variant of the human lipoprotein lipase gene for the treatment of patients with lipoprotein lipase deficiency.

21 January 2018

1 reference

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Premature Atherosclerosis in Patients with Familial Chylomicronemia Caused by Mutations in the Lipoprotein Lipase Gene

28 October 2018

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Comparison of different treatment modalities in experimental pancreatitis in rats

21 January 2018

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12 December 2020

Immunohistological alterations in muscle of patients with amyotrophic lateral sclerosis: mononuclear cell phenotypes and expression of MHC products

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12 December 2020

Acute pancreatitis with hyperlipemia: studies with an isolated perfused canine pancreas

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12 December 2020

Clinical assessment of hyperlipidemic pancreatitis

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12 December 2020

Role of hypertriglyceridemia in the pathogenesis of experimental acute pancreatitis in rats

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12 December 2020

Isolated rat pancreatic acini as a model to study the potential role of lipase in the pathogenesis of acinar cell destruction

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12 December 2020

Etiology and risk of lactescent plasma and severe hypertriglyceridemia

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12 December 2020

Effect of alipogene tiparvovec (AAV1-LPL(S447X)) on postprandial chylomicron metabolism in lipoprotein lipase-deficient patients

1 reference

https://api.crossref.org/works/10.1038%2FGT.2012.43

7 January 2021

inferred from DOI database lookup

Identifiers

DOI

10.1038/GT.2012.43

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Dimensions Publication ID

1 August 2017

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1 August 2017

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