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Selection and evaluation of clinically relevant AAV variants in a xenograft liver model

Nature (London), 2014-02, Vol.506 (7488), p.382-386

2014

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Phase 1–2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B

The New England journal of medicine, 2022-07, Vol.387 (3), p.237-247

2022

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Bioengineered AAV Capsids with Combined High Human Liver Transduction In Vivo and Unique Humoral Seroreactivity

Molecular therapy, 2018-01, Vol.26 (1), p.289-303

2018

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Identification of liver-specific enhancer-promoter activity in the 3' untranslated region of the wild-type AAV2 genome

Nature genetics, 2017-08, Vol.49 (8), p.1267-1273

2017

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Gene Delivery to the Juvenile Mouse Liver Using AAV2/8 Vectors

Molecular therapy, 2008-06, Vol.16 (6), p.1081-1088

2008

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Potential limits of AAV‐based gene therapy with the use of new transgenes expressing factor IX fusion proteins

Haemophilia : the official journal of the World Federation of Hemophilia, 2019-01, Vol.25 (1), p.e11-e18

2019

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Adeno-associated Virus-mediated Rescue of Neonatal Lethality in Argininosuccinate Synthetase-deficient Mice

Molecular therapy, 2013-10, Vol.21 (10), p.1823-1831

2013

Volltextzugriff (PDF)

Induction and Prevention of Severe Hyperammonemia in the spfash Mouse Model of Ornithine Transcarbamylase Deficiency Using shRNA and rAAV-mediated Gene Delivery

Molecular therapy, 2011-05, Vol.19 (5), p.854-859

2011

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Partial pancreatic transdifferentiation of primary human hepatocytes in the livers of a humanised mouse model

The journal of gene medicine, 2018-05, Vol.20 (5), p.e3017-n/a

2018

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Lymphomagenesis in SCID-X1 Mice Following Lentivirus-mediated Phenotype Correction Independent of Insertional Mutagenesis and γc Overexpression

Molecular therapy, 2010-05, Vol.18 (5), p.965-976

2010

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Sexually Dimorphic Patterns of Episomal rAAV Genome Persistence in the Adult Mouse Liver and Correlation With Hepatocellular Proliferation

Molecular therapy, 2009-09, Vol.17 (9), p.1548-1554

2009

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Gene therapy for metabolic disorders: an overview with a focus on urea cycle disorders

Journal of inherited metabolic disease, 2012-07, Vol.35 (4), p.641-645

2012

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Enhancing FVIII Expression By Modifying B-Domain Sequence for Hemophilia A Gene Therapy

Blood, 2018-11, Vol.132 (Supplement 1), p.3484-3484

2018

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Adeno-Associated Virus Mediated Gene Transfer with Hyperfunctional FIX-E410H Variant Improves Mean Factor IX Activity Levels in Hemophilia B Mice

Blood, 2017-12, Vol.130, p.4610-4610

2017

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Preclinical Evaluation of an Engineered AAV Capsid in Non-Human Primates for the Treatment of Haemophilia B

Blood, 2018-11, Vol.132 (Supplement 1), p.2197-2197

2018

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Gene-directed enzyme prodrug therapy for prostate cancer in a mouse model that imitates the development of human disease

The journal of gene medicine, 2004-01, Vol.6 (1), p.43-54

2004

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A Single Intravenous Infusion of FLT180a Results in Factor IX Activity Levels of More Than 40% and Has the Potential to Provide a Functional Cure for Patients with Haemophilia B

Blood, 2018-11, Vol.132 (Supplement 1), p.631-631

2018

Volltextzugriff (PDF)

Purine nucleoside phosphorylase and fludarabine phosphate gene-directed enzyme prodrug therapy suppresses primary tumour growth and pseudo-metastases in a mouse model of prostate cancer

The journal of gene medicine, 2004-12, Vol.6 (12), p.1343-1357

2004

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Liver-Directed AAV Gene Therapy for Gaucher Disease

Blood, 2019-11, Vol.134 (Supplement_1), p.3354-3354

2019

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One-off liver directed AAV gene therapy achieves long term uptake of acid beta-glucocerebrosidase by macrophages of affected tissues in Gaucher disease

Molecular genetics and metabolism, 2020-02, Vol.129 (2), p.S110-S110

2020

Volltextzugriff (PDF)

298. Characterization of a Novel Anti-Sense Promoter Element in Adeno-Associated Virus: Implications for Hepatocellular Carcinoma

Molecular therapy, 2016-05, Vol.24, p.S119-S120

2016

Volltextzugriff (PDF)

Induction and Prevention of Severe Hyperammonemia in the spf super(ash) Mouse Model of Ornithine Transcarbamylase Deficiency Using shRNA and rAAV-mediated Gene Delivery

Molecular therapy, 2011-05, Vol.19 (5), p.854-859

2011

Volltextzugriff (PDF)

Lymphomagenesis in SCID-X1 Mice Following Lentivirus-mediated Phenotype Correction Independent of Insertional Mutagenesis and [gamma]c Overexpression

Molecular therapy, 2010-05, Vol.18 (5), p.965

2010

Volltextzugriff (PDF)

Application of the transgenic adenocarcinoma mouse prostate (TRAMP) model for pre-clinical therapeutic studies

Anticancer research, 2003-05, Vol.23 (3B), p.2633

2003

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Gene Delivery to the Juvenile Mouse Liver Using AAV2/8 Vectors

Molecular therapy, 2008-06, Vol.16 (6), p.1081

2008

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Selection and evaluation of clinically relevant AAV variants in a xenograft liver model

Phase 1–2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B

Bioengineered AAV Capsids with Combined High Human Liver Transduction In Vivo and Unique Humoral Seroreactivity

Identification of liver-specific enhancer-promoter activity in the 3' untranslated region of the wild-type AAV2 genome

Gene Delivery to the Juvenile Mouse Liver Using AAV2/8 Vectors

Potential limits of AAV‐based gene therapy with the use of new transgenes expressing factor IX fusion proteins

Adeno-associated Virus-mediated Rescue of Neonatal Lethality in Argininosuccinate Synthetase-deficient Mice

Induction and Prevention of Severe Hyperammonemia in the spfash Mouse Model of Ornithine Transcarbamylase Deficiency Using shRNA and rAAV-mediated Gene Delivery

Partial pancreatic transdifferentiation of primary human hepatocytes in the livers of a humanised mouse model

Lymphomagenesis in SCID-X1 Mice Following Lentivirus-mediated Phenotype Correction Independent of Insertional Mutagenesis and γc Overexpression

Sexually Dimorphic Patterns of Episomal rAAV Genome Persistence in the Adult Mouse Liver and Correlation With Hepatocellular Proliferation

Gene therapy for metabolic disorders: an overview with a focus on urea cycle disorders

Enhancing FVIII Expression By Modifying B-Domain Sequence for Hemophilia A Gene Therapy

Adeno-Associated Virus Mediated Gene Transfer with Hyperfunctional FIX-E410H Variant Improves Mean Factor IX Activity Levels in Hemophilia B Mice

Preclinical Evaluation of an Engineered AAV Capsid in Non-Human Primates for the Treatment of Haemophilia B

Gene-directed enzyme prodrug therapy for prostate cancer in a mouse model that imitates the development of human disease

A Single Intravenous Infusion of FLT180a Results in Factor IX Activity Levels of More Than 40% and Has the Potential to Provide a Functional Cure for Patients with Haemophilia B

Purine nucleoside phosphorylase and fludarabine phosphate gene-directed enzyme prodrug therapy suppresses primary tumour growth and pseudo-metastases in a mouse model of prostate cancer

Liver-Directed AAV Gene Therapy for Gaucher Disease

One-off liver directed AAV gene therapy achieves long term uptake of acid beta-glucocerebrosidase by macrophages of affected tissues in Gaucher disease

298. Characterization of a Novel Anti-Sense Promoter Element in Adeno-Associated Virus: Implications for Hepatocellular Carcinoma

Induction and Prevention of Severe Hyperammonemia in the spf super(ash) Mouse Model of Ornithine Transcarbamylase Deficiency Using shRNA and rAAV-mediated Gene Delivery

Lymphomagenesis in SCID-X1 Mice Following Lentivirus-mediated Phenotype Correction Independent of Insertional Mutagenesis and [gamma]c Overexpression

Application of the transgenic adenocarcinoma mouse prostate (TRAMP) model for pre-clinical therapeutic studies

Gene Delivery to the Juvenile Mouse Liver Using AAV2/8 Vectors

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