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State of the art: gene therapy of haemophilia

Haemophilia : the official journal of the World Federation of Hemophilia, 2016-07, Vol.22 (S5), p.66-71

2016

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Generation of an optimized lentiviral vector encoding a high-expression factor VIII transgene for gene therapy of hemophilia A

Gene therapy, 2013-06, Vol.20 (6), p.607-615

2013

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Expanding the ortholog approach for hemophilia treatment complicated by factor VIII inhibitors

Journal of thrombosis and haemostasis, 2015-01, Vol.13 (1), p.72-81

2015

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High-throughput screening identifies compounds that enhance lentiviral transduction

Gene therapy, 2014-12, Vol.21 (12), p.1008-1020

2014

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High Level Expression of Recombinant Porcine Coagulation Factor VIII

The Journal of biological chemistry, 2002-10, Vol.277 (41), p.38345-38349

2002

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Amino acid deprivation induces translation of branched-chain alpha -ketoacid dehydrogenase kinase

American Journal of Physiology: Cell Physiology, 2000-11, Vol.279 (5), p.C1587-C1594

2000

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High-dose factor VIII inhibits factor VIII–specific memory B cells in hemophilia A with factor VIII inhibitors

Blood, 2005-11, Vol.106 (10), p.3415-3422

2005

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Murine branched chain α-ketoacid dehydrogenase kinase; cDNA cloning, tissue distribution, and temporal expression during embryonic development

Gene, 1998-06, Vol.212 (2), p.213-219

1998

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Expression of murine branched-chain α-keto acid dehydrogenase kinase

Methods in Enzymology, 2000, Vol.324, p.491-497

2000

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Leucine toxicity in a neuronal cell model with inhibited branched chain amino acid catabolism

Brain research. Molecular brain research., 2004-03, Vol.122 (2), p.180-187

2004

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Expression and characterization of recombinant murine factor VIII

Thrombosis and haemostasis, 2002-09, Vol.88 (3), p.450-458

2002

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Human mutations affecting branched chain alpha-ketoacid dehydrogenase

Frontiers in bioscience, 1998-06, Vol.3, p.d517

1998

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Insulin increases branched-chain alpha -ketoacid dehydrogenase kinase expression in Clone 9 rat cells

American journal of physiology: endocrinology and metabolism, 2002-10, Vol.283 (4), p.E853-E860

2002

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Factor VIII expression in azoxymethane-induced murine fulminant hepatic failure

Blood, 2002-07, Vol.100 (1), p.143-147

2002

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Decreased factor VIII levels during acetaminophen-induced murine fulminant hepatic failure

Blood, 2003-09, Vol.102 (5), p.1743-1744

2003

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Controlled Overexpression of BCKD Kinase Expression: Metabolic Engineering Applied to BCAA Metabolism in a Mammalian System

Metabolic engineering, 2000-10, Vol.2 (4), p.349-356

2000

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Identifying the risk of obstructive sleep apnea in metabolic syndrome patients: Diagnostic accuracy of the Berlin Questionnaire

PloS one, 2019-05, Vol.14 (5), p.e0217058

2019

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Development and psychometric evaluation of the Decision Tool Anxiety Disorders, OCD and PTSD (DTAOP): Facilitating the early detection of patients in need of highly specialized care

PloS one, 2021-01, Vol.16 (8), p.e0256384

2021

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A web-survey assessed attitudes toward evidence-based practice among psychotherapists in Austria

Scientific reports, 2022-06, Vol.12 (1), p.9374-9374, Article 9374

2022

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Transient B cell depletion or improved transgene expression by codon optimization promote tolerance to factor VIII in gene therapy

PloS one, 2012-05, Vol.7 (5), p.e37671-e37671

2012

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Enhancing the pharmaceutical properties of protein drugs by ancestral sequence reconstruction

Nature biotechnology, 2017-01, Vol.35 (1), p.35-37

2017

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Why Metrics Matter: Evaluating Policy Choices for Reactive Nitrogen in the Chesapeake Bay Watershed

Environmental science & technology, 2011-01, Vol.45 (1), p.168-174

2011

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Author Correction: A web-survey assessed attitudes toward evidence-based practice among psychotherapists in Austria

Scientific reports, 2022-08, Vol.12 (1), p.13683-13683, Article 13683

2022

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Characterization of coding synonymous and non-synonymous variants in ADAMTS13 using ex vivo and in silico approaches

PloS one, 2012-06, Vol.7 (6), p.e38864

2012

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Platelet clearance via shear-induced unfolding of a membrane mechanoreceptor

Nature communications, 2016-09, Vol.7 (1), p.12863-12863, Article 12863

2016

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State of the art: gene therapy of haemophilia

Generation of an optimized lentiviral vector encoding a high-expression factor VIII transgene for gene therapy of hemophilia A

Expanding the ortholog approach for hemophilia treatment complicated by factor VIII inhibitors

High-throughput screening identifies compounds that enhance lentiviral transduction

High Level Expression of Recombinant Porcine Coagulation Factor VIII

Amino acid deprivation induces translation of branched-chain alpha -ketoacid dehydrogenase kinase

High-dose factor VIII inhibits factor VIII–specific memory B cells in hemophilia A with factor VIII inhibitors

Murine branched chain α-ketoacid dehydrogenase kinase; cDNA cloning, tissue distribution, and temporal expression during embryonic development

Expression of murine branched-chain α-keto acid dehydrogenase kinase

Leucine toxicity in a neuronal cell model with inhibited branched chain amino acid catabolism

Expression and characterization of recombinant murine factor VIII

Human mutations affecting branched chain alpha-ketoacid dehydrogenase

Insulin increases branched-chain alpha -ketoacid dehydrogenase kinase expression in Clone 9 rat cells

Factor VIII expression in azoxymethane-induced murine fulminant hepatic failure

Decreased factor VIII levels during acetaminophen-induced murine fulminant hepatic failure

Controlled Overexpression of BCKD Kinase Expression: Metabolic Engineering Applied to BCAA Metabolism in a Mammalian System

Identifying the risk of obstructive sleep apnea in metabolic syndrome patients: Diagnostic accuracy of the Berlin Questionnaire

Development and psychometric evaluation of the Decision Tool Anxiety Disorders, OCD and PTSD (DTAOP): Facilitating the early detection of patients in need of highly specialized care

A web-survey assessed attitudes toward evidence-based practice among psychotherapists in Austria

Transient B cell depletion or improved transgene expression by codon optimization promote tolerance to factor VIII in gene therapy

Enhancing the pharmaceutical properties of protein drugs by ancestral sequence reconstruction

Why Metrics Matter: Evaluating Policy Choices for Reactive Nitrogen in the Chesapeake Bay Watershed

Author Correction: A web-survey assessed attitudes toward evidence-based practice among psychotherapists in Austria

Characterization of coding synonymous and non-synonymous variants in ADAMTS13 using ex vivo and in silico approaches

Platelet clearance via shear-induced unfolding of a membrane mechanoreceptor

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