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Gene therapy for hemophilia

Journal of thrombosis and haemostasis, 2013-06, Vol.11, p.99-110

2013

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Next-generation muscle-directed gene therapy by in silico vector design

Nature communications, 2019-01, Vol.10 (1), p.492-492, Article 492

2019

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Therapeutic Approaches for Dominant Muscle Diseases: Highlight on Myotonic Dystrophy

Current gene therapy, 2015-01, Vol.15 (4), p.329-337

2015

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Efficacy and safety of adeno‐associated viral vectors based on serotype 8 and 9 vs. lentiviral vectors for hemophilia B gene therapy

Journal of thrombosis and haemostasis, 2007-01, Vol.5 (1), p.16-24

2007

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Clinical progress in gene therapy: sustained partial correction of the bleeding disorder in patients suffering from severe hemophilia B

Human gene therapy, 2012-01, Vol.23 (1), p.4-6

2012

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Platelet‐directed gene therapy overcomes inhibitory antibodies to factor VIII

Journal of thrombosis and haemostasis, 2012-08, Vol.10 (8), p.1566-1569

2012

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Gene therapy for the hemophilias

Journal of thrombosis and haemostasis, 2003-07, Vol.1 (7), p.1550-1558

2003

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Harmonic analysis of compact Lie supergroups

Expositiones mathematicae, 2024-07, p.125586, Article 125586

2024

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Liver-directed lentiviral gene therapy in a dog model of hemophilia B

Science translational medicine, 2015-03, Vol.7 (277), p.277ra28

2015

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Long-term persistence of human bone marrow stromal cells transduced with factor VIII-retroviral vectors and transient production of therapeutic levels of human factor VIII in nonmyeloablated immunodeficient mice

Human gene therapy, 2000-03, Vol.11 (5), p.729-738

2000

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Preclinical and clinical gene therapy for haemophilia

Haemophilia : the official journal of the World Federation of Hemophilia, 2004-10, Vol.10 (s4), p.119-125

2004

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Liver-Specific Transcriptional Modules Identified by Genome-Wide In Silico Analysis Enable Efficient Gene Therapy in Mice and Non-Human Primates

Molecular therapy, 2014-09, Vol.22 (9), p.1605-1613

2014

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Recent progress in gene therapy for hemophilia

Human gene therapy, 2012-06, Vol.23 (6), p.557-565

2012

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Bone marrow mesenchymal cells for haemophilia A gene therapy using retroviral vectors with modified long-terminal repeats

Haemophilia : the official journal of the World Federation of Hemophilia, 2003-01, Vol.9 (1), p.94-103

2003

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Vector decoys trick the immune response

Science translational medicine, 2013-07, Vol.5 (194), p.194fs28

2013

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Genetic aspects and research development in haemostasis

Haemophilia : the official journal of the World Federation of Hemophilia, 2008-07, Vol.14 (s3), p.113-118

2008

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Viral vector-mediated gene therapy for hemophilia

Current gene therapy, 2001-09, Vol.1 (3), p.301-315

2001

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Inhibition of clinical human immunodeficiency virus (HIV) type 1 isolates in primary CD4+ T lymphocytes by retroviral vectors expressing anti-HIV genes

Journal of Virology, 1995-07, Vol.69 (7), p.4045-4052

1995

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Gene therapy for hemophilia: hopes and hurdles

Critical reviews in oncology/hematology, 1998-09, Vol.28 (3), p.153-171

1998

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Gene therapy for haemophilia "A" and "B": efficacy, safety and immune consequences

Bulletin et memoires de l'Academie royale de medecine de Belgique, 2007, Vol.162 (5-6), p.357

2007

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Long-Term Expression of Human Coagulation Factor VIII and Correction of Hemophilia A after in vivo Retroviral Gene Transfer in Factor VIII-Deficient Mice

Proceedings of the National Academy of Sciences - PNAS, 1999-08, Vol.96 (18), p.10379-10384

1999

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A novel cause of mild/moderate hemophilia A : mutations scattered in the factor VIII C1 domain reduce factor VIII binding to von Willebrand factor

Blood, 2000-08, Vol.96 (3), p.958-965

2000

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Gene therapy for hemophilia

The journal of gene medicine, 2001-01, Vol.3 (1), p.3-20

2001

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Bone marrow stromal cells as targets for gene therapy of hemophilia A

Human gene therapy, 1998-02, Vol.9 (3), p.353-365

1998

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Equivalence classes of Vogan diagrams

Journal of algebra, 2004-09, Vol.279 (1), p.22-37

2004

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Gene therapy for hemophilia

Next-generation muscle-directed gene therapy by in silico vector design

Therapeutic Approaches for Dominant Muscle Diseases: Highlight on Myotonic Dystrophy

Efficacy and safety of adeno‐associated viral vectors based on serotype 8 and 9 vs. lentiviral vectors for hemophilia B gene therapy

Clinical progress in gene therapy: sustained partial correction of the bleeding disorder in patients suffering from severe hemophilia B

Platelet‐directed gene therapy overcomes inhibitory antibodies to factor VIII

Gene therapy for the hemophilias

Harmonic analysis of compact Lie supergroups

Liver-directed lentiviral gene therapy in a dog model of hemophilia B

Long-term persistence of human bone marrow stromal cells transduced with factor VIII-retroviral vectors and transient production of therapeutic levels of human factor VIII in nonmyeloablated immunodeficient mice

Preclinical and clinical gene therapy for haemophilia

Liver-Specific Transcriptional Modules Identified by Genome-Wide In Silico Analysis Enable Efficient Gene Therapy in Mice and Non-Human Primates

Recent progress in gene therapy for hemophilia

Bone marrow mesenchymal cells for haemophilia A gene therapy using retroviral vectors with modified long-terminal repeats

Vector decoys trick the immune response

Genetic aspects and research development in haemostasis

Viral vector-mediated gene therapy for hemophilia

Inhibition of clinical human immunodeficiency virus (HIV) type 1 isolates in primary CD4+ T lymphocytes by retroviral vectors expressing anti-HIV genes

Gene therapy for hemophilia: hopes and hurdles

Gene therapy for haemophilia "A" and "B": efficacy, safety and immune consequences

Long-Term Expression of Human Coagulation Factor VIII and Correction of Hemophilia A after in vivo Retroviral Gene Transfer in Factor VIII-Deficient Mice

A novel cause of mild/moderate hemophilia A : mutations scattered in the factor VIII C1 domain reduce factor VIII binding to von Willebrand factor

Gene therapy for hemophilia

Bone marrow stromal cells as targets for gene therapy of hemophilia A

Equivalence classes of Vogan diagrams

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