Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial (Q34311144)

1 August 2017

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

1 reference

1 August 2017

0 references

D Gaudet

1

1 reference

1 August 2017

J Méthot

2

1 reference

1 August 2017

S Déry

3

1 reference

1 August 2017

D Brisson

4

1 reference

1 August 2017

C Essiembre

5

1 reference

1 August 2017

G Tremblay

6

1 reference

1 August 2017

K Tremblay

7

1 reference

1 August 2017

J de Wal

8

1 reference

1 August 2017

J Twisk

9

1 reference

1 August 2017

N van den Bulk

10

1 reference

1 August 2017

V Sier-Ferreira

11

1 reference

1 August 2017

S van Deventer

12

1 reference

1 August 2017

language of work or name

1 reference

21 June 2012

1 reference

1 August 2017

1 reference

1 August 2017

20

1 reference

1 August 2017

4

1 reference

1 August 2017

361-369

1 reference

https://scigraph.springernature.com/pub.10.1038/gt.2012.43

1 August 2017

0 references

cites work

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4956470

Comparison of the efficacy of fibrates on hypertriglyceridemic phenotypes with different genetic and clinical characteristics

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4956470

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

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4956470

Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4956470

AAV-1-mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4956470

Infectious molecular clones of adeno-associated virus isolated directly from human tissues

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4956470

Gene therapy using adeno-associated virus vectors

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4956470

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

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4956470

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

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4956470

Lipoprotein lipase: structure, function, regulation, and role in disease

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4956470

Remnant lipoprotein metabolism: key pathways involving cell-surface heparan sulfate proteoglycans and apolipoprotein E

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4956470

The familial chylomicronemia syndrome

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4956470

Lipoprotein lipase and lipolysis: central roles in lipoprotein metabolism and atherogenesis.

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4956470

Dietary treatment and growth of hyperchylomicronemic children severely restricted in dietary fat.

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4956470

Chylomicronemia syndrome in diabetes mellitus.

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4956470

Human adipose tissue lipoprotein lipase: changes with feeding and relation to postheparin plasma enzyme

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4956470

Lymphocyte activation markers in idiopathic myositis: changes with disease activity and differences among clinical and autoantibody subgroups

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4956470

Chylomicronemia syndrome.

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4956470

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

6 July 2018

1 reference

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

Gene therapy for lipoprotein lipase deficiency: working toward clinical application

21 January 2018

1 reference

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

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

1 reference

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

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

21 January 2018

1 reference

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

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

21 January 2018

1 reference

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

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

21 January 2018

1 reference

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

Congenital disorder of glycosylation type Ia: a clinicopathological report of a newborn infant with cerebellar pathology

21 January 2018

1 reference

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

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

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4956470

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

28 October 2018

1 reference

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

Comparison of different treatment modalities in experimental pancreatitis in rats

21 January 2018

1 reference

12 December 2020

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

1 reference

12 December 2020

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

1 reference

12 December 2020

Clinical assessment of hyperlipidemic pancreatitis

1 reference

12 December 2020

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

1 reference

12 December 2020

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

1 reference

12 December 2020

Etiology and risk of lactescent plasma and severe hypertriglyceridemia

1 reference

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

1 reference

Dimensions Publication ID

1 August 2017

0 references

1 reference

1 August 2017

1 reference