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BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expression

Blood, 2021-02, Vol.137 (6), p.763-774

2021

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Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector

Molecular therapy, 2012-02, Vol.20 (2), p.443-455

2012

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Prophylactic efficacy of BeneFIX vs Alprolix in hemophilia B mice

Blood, 2016-07, Vol.128 (2), p.286-292

2016

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Employing a gain-of-function factor IX variant R338L to advance the efficacy and safety of hemophilia B human gene therapy: preclinical evaluation supporting an ongoing adeno-associated virus clinical trial

Human gene therapy, 2015-02, Vol.26 (2), p.69-81

2015

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Single Amino Acid Modification of Adeno-Associated Virus Capsid Changes Transduction and Humoral Immune Profiles

Journal of Virology, 2012-08, Vol.86 (15), p.7752-7759

2012

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Autologous and Heterologous Cell Therapy for Hemophilia B toward Functional Restoration of Factor IX

Cell reports (Cambridge), 2018-05, Vol.23 (5), p.1565-1580

2018

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Epigenetic activation of unintegrated HIV-1 genomes by gut-associated short chain fatty acids and its implications for HIV infection

Proceedings of the National Academy of Sciences - PNAS, 2009-11, Vol.106 (44), p.18786-18791

2009

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The frequency of joint hemorrhages and procedures in nonsevere hemophilia A vs B

Blood advances, 2018-08, Vol.2 (16), p.2136-2144

2018

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Proteasome Inhibitors Enhance Gene Delivery by AAV Virus Vectors Expressing Large Genomes in Hemophilia Mouse and Dog Models: A Strategy for Broad Clinical Application

Molecular therapy, 2010-11, Vol.18 (11), p.1907-1916

2010

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Optimization of Self-complementary AAV Vectors for Liver-directed Expression Results in Sustained Correction of Hemophilia B at Low Vector Dose

Molecular therapy, 2008-02, Vol.16 (2), p.280-289

2008

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Comprehensive analysis and prediction of long-term durability of factor IX activity following etranacogene dezaparvovec gene therapy in the treatment of hemophilia B

Current medical research and opinion, 2023-02, Vol.39 (2), p.227-237

2023

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Physical Functioning in Boys with Hemophilia in the U.S

American journal of preventive medicine, 2011-12, Vol.41 (6), p.S360-S368

2011

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Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A

The New England journal of medicine, 2021-11, Vol.385 (21), p.1961-1973

2021

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Long-Term Effects of Hemophilia B Gene Therapy. Reply

The New England journal of medicine, 2023-05, Vol.388 (20), p.1918-1919

2023

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An Observational Study from Long-Term AAV Re-administration in Two Hemophilia Dogs

Molecular therapy. Methods & clinical development, 2018-09, Vol.10, p.257-267

2018

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Intraarticular factor IX protein or gene replacement protects against development of hemophilic synovitis in the absence of circulating factor IX

Blood, 2008-12, Vol.112 (12), p.4532-4541

2008

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Development of Methods for the Selective Measurement of the Single Amino Acid Exchange Variant Coagulation Factor IX Padua

Molecular therapy. Methods & clinical development, 2018-09, Vol.10, p.29-37

2018

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Emerging genetic and pharmacologic therapies for controlling hemostasis: beyond recombinant clotting factors

Hematology, 2015-12, Vol.2015 (1), p.33-40

Monahan, Paul E

2015

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Factor IX: Insights from knock-out and genetically engineered mice

Thrombosis and haemostasis, 2008-10, Vol.100 (4), p.563-575

Monahan, Paul E

2008

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Men with severe hemophilia in the United States: birth cohort analysis of a large national database

Blood, 2016-06, Vol.127 (24), p.3073-3081

2016

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Emerging Immunogenicity and Genotoxicity Considerations of Adeno-Associated Virus Vector Gene Therapy for Hemophilia

Journal of clinical medicine, 2021-06, Vol.10 (11), p.2471

2021

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FVIII at the crossroad of coagulation, bone and immune biology: Emerging evidence of biological activities beyond hemostasis

Drug discovery today, 2022-01, Vol.27 (1), p.102-116

2022

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Exploring the Potential Feasibility of Intra-Articular Adeno-Associated Virus-Mediated Gene Therapy for Hemophilia Arthropathy

Human gene therapy, 2020-04, Vol.31 (7-8), p.448-458

2020

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Indirect treatment comparisons of the gene therapy etranacogene dezaparvovec versus extended half‐life factor IX therapies for severe or moderately severe haemophilia B

Haemophilia : the official journal of the World Federation of Hemophilia, 2024-01, Vol.30 (1), p.75-86

2024

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Discussing investigational AAV gene therapy with hemophilia patients: A guide

Blood reviews, 2021-05, Vol.47, p.100759-100759, Article 100759

2021

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BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expression

Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector

Prophylactic efficacy of BeneFIX vs Alprolix in hemophilia B mice

Employing a gain-of-function factor IX variant R338L to advance the efficacy and safety of hemophilia B human gene therapy: preclinical evaluation supporting an ongoing adeno-associated virus clinical trial

Single Amino Acid Modification of Adeno-Associated Virus Capsid Changes Transduction and Humoral Immune Profiles

Autologous and Heterologous Cell Therapy for Hemophilia B toward Functional Restoration of Factor IX

Epigenetic activation of unintegrated HIV-1 genomes by gut-associated short chain fatty acids and its implications for HIV infection

The frequency of joint hemorrhages and procedures in nonsevere hemophilia A vs B

Proteasome Inhibitors Enhance Gene Delivery by AAV Virus Vectors Expressing Large Genomes in Hemophilia Mouse and Dog Models: A Strategy for Broad Clinical Application

Optimization of Self-complementary AAV Vectors for Liver-directed Expression Results in Sustained Correction of Hemophilia B at Low Vector Dose

Comprehensive analysis and prediction of long-term durability of factor IX activity following etranacogene dezaparvovec gene therapy in the treatment of hemophilia B

Physical Functioning in Boys with Hemophilia in the U.S

Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A

Long-Term Effects of Hemophilia B Gene Therapy. Reply

An Observational Study from Long-Term AAV Re-administration in Two Hemophilia Dogs

Intraarticular factor IX protein or gene replacement protects against development of hemophilic synovitis in the absence of circulating factor IX

Development of Methods for the Selective Measurement of the Single Amino Acid Exchange Variant Coagulation Factor IX Padua

Emerging genetic and pharmacologic therapies for controlling hemostasis: beyond recombinant clotting factors

Factor IX: Insights from knock-out and genetically engineered mice

Men with severe hemophilia in the United States: birth cohort analysis of a large national database

Emerging Immunogenicity and Genotoxicity Considerations of Adeno-Associated Virus Vector Gene Therapy for Hemophilia

FVIII at the crossroad of coagulation, bone and immune biology: Emerging evidence of biological activities beyond hemostasis

Exploring the Potential Feasibility of Intra-Articular Adeno-Associated Virus-Mediated Gene Therapy for Hemophilia Arthropathy

Indirect treatment comparisons of the gene therapy etranacogene dezaparvovec versus extended half‐life factor IX therapies for severe or moderately severe haemophilia B

Discussing investigational AAV gene therapy with hemophilia patients: A guide

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