Gene therapy for the hemophilias

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Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector

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In Vivo Gene Therapy of Hemophilia B: Sustained Partial Correction in Factor IX-Deficient Dogs

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Plasma Throinboplastin Component (PTC) Deficiency: A New Disease Resembling Hemophilia

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In vivo hepatic gene therapy: complete albeit transient correction of factor IX deficiency in hemophilia B dogs.

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Sustained correction of bleeding disorder in hemophilia B mice by gene therapy

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Canine hemophilia B resulting from a point mutation with unusual consequences.

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Production of human factor IX in animals by genetically modified skin fibroblasts: potential therapy for hemophilia B

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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

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A factor IX-deficient mouse model for hemophilia B gene therapy

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Sequences in the Coding Region of Clotting Factor VIII Act as Dominant Inhibitors of RNA Accumulation and Protein Production

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Pseudotransduction of hepatocytes by using concentrated pseudotyped vesicular stomatitis virus G glycoprotein (VSV-G)-Moloney murine leukemia virus-derived retrovirus vectors: comparison of VSV-G and amphotropic vectors for hepatic gene transfer

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Expression of human factor IX in rat capillary endothelial cells: toward somatic gene therapy for hemophilia B.

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Hepatocyte growth factor induces hepatocyte proliferation in vivo and allows for efficient retroviral-mediated gene transfer in mice

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Gene therapy for hemophilia A: production of therapeutic levels of human factor VIII in vivo in mice.

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Genetic induction of immune tolerance to human clotting factor VIII in a mouse model for hemophilia A

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A deletion mutation causes hemophilia B in Lhasa Apso dogs

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Targeted Inactivation of the Coagulation Factor IX Gene Causes Hemophilia B in Mice

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Proliferation induced by keratinocyte growth factor enhances in vivo retroviral-mediated gene transfer to mouse hepatocytes.

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The Canine Factor VIII cDNA and 5’ Flanking Sequence

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Antigen Presentation – Recent Developments

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WHY DO SO MANY HAEMOPHILIA A PATIENTS DEVELOP AN INHIBITOR?

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Genetics and molecular biology of haemophilias A and B

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Factors impeding efficient expression of factor VIII complementary DNA minigenes.

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Gene transfer by lentiviral vectors is limited by nuclear translocation and rescued by HIV-1 pol sequences

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AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B

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Production of gene-targeted sheep by nuclear transfer from cultured somatic cells

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Efficient Gene Delivery and Targeted Expression to Hepatocytes In Vivo by Improved Lentiviral Vectors

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Kinetics of Liver Repopulation after Bone Marrow Transplantation

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Preclinical in vivo evaluation of pseudotyped adeno-associated virus vectors for liver gene therapy

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Muscle-Directed Gene Transfer and Transient Immune Suppression Result in Sustained Partial Correction of Canine Hemophilia B Caused by a Null Mutation

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Sustained Expression of Therapeutic Level of Factor IX in Hemophilia B Dogs by AAV-Mediated Gene Therapy in Liver

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Use of blood outgrowth endothelial cells for gene therapy for hemophilia A

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Therapeutic factor VIII levels and negligible toxicity in mouse and dog models of hemophilia A following gene therapy with high-capacity adenoviral vectors

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Phenotypic correction and long-term expression of factor VIII in hemophilic mice by immunotolerization and nonviral gene transfer using the Sleeping Beauty transposon system

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Adeno‐associated virus gene repair corrects a mouse model of hereditary tyrosinemia in vivo†‡

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Home management of haemophilia

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Understanding hereditary diseases using the dog and human as companion model systems

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Ex Vivo Gene Therapy for Hemophilia A That Enhances Safe Delivery and Sustained In Vivo Factor VIII Expression from Lentivirally Engineered Endothelial Progenitors

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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

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Nonviral Approaches Satisfying Various Requirements for Effective in Vivo Gene Therapy.

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Sustained Whole-Body Functional Rescue in Congestive Heart Failure and Muscular Dystrophy Hamsters by Systemic Gene Transfer

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In utero stem cell transplantation and gene therapy: rationale, history, and recent advances toward clinical application

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Gene therapy for hemophilia

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Efficacy of engineered FVIII-producing skeletal muscle enhanced by growth factor-releasing co-axial electrospun fibers

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Gene Transfer as an Approach to Treating Hemophilia

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Gene therapy for the hemophilias

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AAV‐Mediated Gene Transfer for Hemophilia

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Complete Correction of Hemophilia A with Adeno-Associated Viral Vectors Containing a Full-Size Expression Cassette

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Hepatic insulin expression improves glycemic control in type 1 diabetic rats

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AAV-based neonatal gene therapy for hemophilia A: long-term correction and avoidance of immune responses in mice

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Gene therapy strategies for hemophilia: benefits versus risks

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The Enhancing Effects of the Light Chain on Heavy Chain Secretion in Split Delivery of Factor VIII Gene

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Dual Vectors Expressing Murine Factor VIII Result in Sustained Correction of Hemophilia A Mice

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AAV-Mediated Gene Targeting Is Significantly Enhanced by Transient Inhibition of Nonhomologous End Joining or the Proteasome In Vivo

Paulk, N. K., Loza, L. I. M., Finegold, M. J., & Grompe, M. (2012) · Human Gene Therapy · doi:10.1089/hum.2012.038 · Cited by 35 · see citing works

Bone marrow stromal cells as a genetic platformfor systemic delivery of therapeutic proteins in vivo: human factor IX model

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Congenital Hemorrhagic Disorders: New Insights into the Pathophysiology and Treatment of Hemophilia

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The Canine Factor VIII 3′-Untranslated Region and a Concatemeric Hepatocyte Nuclear Factor 1 Regulatory Element Enhance Factor VIII Transgene Expression In Vivo

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AAV-mediated gene transfer for hemophilia

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FVIII gene delivery by muscle electroporation corrects murine hemophilia A

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Epstein‐Barr Virus Vectors Provide Prolonged Robust Factor IX Expression in Mice

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Adenovirus-Mediated Factor VIII Gene Expression Results in Attenuated Anti-Factor VIII-Specific Immunity in Hemophilia A Mice Compared with Factor VIII Protein Infusion

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Hemophilia A: an ideal disease to correct in utero

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Efficient AAV1–AAV2 Hybrid Vector for Gene Therapy of Hemophilia

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Sustained transgene expression by human cord blood derived CD34+ cells transduced with simian immunodeficiency virus agmTYO1‐based vectors carrying the human coagulation factor VIII gene in NOD/SCID mice

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Gene modification therapies for hereditary diseases in the fetus

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Gene therapy: a 2001 perspective

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