Pathogenesis of Hepatic Tumors following Gene Therapy in Murine and Canine Models of Glycogen Storage Disease

scientific article published on 11 November 2019

Pathogenesis of Hepatic Tumors following Gene Therapy in Murine and Canine Models of Glycogen Storage Disease is …
instance of (P31):
scholarly articleQ13442814

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P356DOI10.1016/J.OMTM.2019.10.016
P932PMC publication ID6909089
P698PubMed publication ID31890731

P2093author name stringFabienne Rajas
Gilles Mithieux
Hye-Ri Kang
Charles P Venditti
Randy J Chandler
Dwight D Koeberl
Songtao Li
Shawn M Burgess
Elizabeth D Brooks
Zelin Chen
Monika Gjorgjieva
Lauren R Waskowicz
Kylie M Grady
Stephanie N Smith
P2860cites workAnalysis of Relative Gene Expression Data Using Real-Time Quantitative PCR and the 2−ΔΔCT MethodQ25938999
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Canine model and genomic structural organization of glycogen storage disease type Ia (GSD Ia).Q30659161
Long-term efficacy following readministration of an adeno-associated virus vector in dogs with glycogen storage disease type IaQ33720634
Complete normalization of hepatic G6PC deficiency in murine glycogen storage disease type Ia using gene therapyQ33930092
Adeno-associated virus-mediated correction of a canine model of glycogen storage disease type Ia.Q34123293
Hepatocellular carcinoma in glycogen storage disease type Ia: a case seriesQ34557393
Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy.Q35056714
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Hepatorenal correction in murine glycogen storage disease type I with a double-stranded adeno-associated virus vectorQ35568513
Induction of hepatocellular carcinoma by in vivo gene targetingQ36094151
Prevention of hepatocellular adenoma and correction of metabolic abnormalities in murine glycogen storage disease type Ia by gene therapyQ36336550
Genotoxicity in Mice Following AAV Gene Delivery: A Safety Concern for Human Gene Therapy?Q36756903
Recombinant adeno-associated virus integration sites in murine liver after ornithine transcarbamylase gene correctionQ36848708
In Vivo Zinc Finger Nuclease-mediated Targeted Integration of a Glucose-6-phosphatase Transgene Promotes Survival in Mice With Glycogen Storage Disease Type IA.Q36951279
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Low-Dose Liver-Targeted Gene Therapy for Pompe Disease Enhances Therapeutic Efficacy of ERT via Immune Tolerance InductionQ37717893
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Gene targeting to the ROSA26 locus directed by engineered zinc finger nucleasesQ39428538
Gene delivery to the juvenile mouse liver using AAV2/8 vectorsQ39991575
Glycogen storage disease type Ia mice with less than 2% of normal hepatic glucose-6-phosphatase-α activity restored are at risk of developing hepatic tumorsQ40376931
Efficacy of an adeno-associated virus 8-pseudotyped vector in glycogen storage disease type II.Q40483404
Autophagy-deficient mice develop multiple liver tumorsQ42099409
Hepatic lentiviral gene transfer prevents the long-term onset of hepatic tumours of glycogen storage disease type 1a in miceQ42175592
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Early, sustained efficacy of adeno-associated virus vector-mediated gene therapy in glycogen storage disease type Ia.Q45417049
AAV vector integration sites in mouse hepatocellular carcinoma.Q45869780
AAV vector-mediated reversal of hypoglycemia in canine and murine glycogen storage disease type Ia.Q45876170
A comparison of whole-genome shotgun-derived mouse chromosome 16 and the human genomeQ48300754
Induction of autophagy improves hepatic lipid metabolism in glucose-6-phosphatase deficiency.Q50924982
Hepatic glucose-6-phosphatase-α deficiency leads to metabolic reprogramming in glycogen storage disease type Ia.Q52726308
Cornstarch regimens for nocturnal treatment of young adults with type I glycogen storage diseaseQ73293457
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Targeted deletion of liver glucose-6 phosphatase mimics glycogen storage disease type 1a including development of multiple adenomasQ82615010
Incidence and natural history of mucopolysaccharidosis type III in France and comparison with United Kingdom and GreeceQ83120890
Long-term safety and efficacy of AAV gene therapy in the canine model of glycogen storage disease type IaQ88868479
Long-term complications of glycogen storage disease type Ia in the canine model treated with gene replacement therapyQ90457188
Dietary exacerbation of metabolic stress leads to accelerated hepatic carcinogenesis in glycogen storage disease type IaQ91334741
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Bezafibrate induces autophagy and improves hepatic lipid metabolism in glycogen storage disease type IaQ91765348
P921main subjectglycogen storage diseaseQ1421738
P1104number of pages9
P304page(s)383-391
P577publication date2019-11-11
P1433published inMolecular Therapy. Methods & Clinical DevelopmentQ27725341
P1476titlePathogenesis of Hepatic Tumors following Gene Therapy in Murine and Canine Models of Glycogen Storage Disease
P478volume15

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