Gene therapy for hemophilia

An induced pluripotent stem cell-mediated and integration-free factor VIII expression system

(2013) Yuwna Yakura et al.   BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS

Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8+ T cells

(2013) A. T. Martino et al.   BLOOD

Hyperfunctional coagulation factor IX improves the efficacy of gene therapy in hemophilic mice

(2012) A. Cantore et al.   BLOOD

Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer

(2012) G. Buchlis et al.   BLOOD

The efficacy and the risk of immunogenicity of FIX Padua (R338L) in hemophilia B dogs treated by AAV muscle gene therapy

(2012) J. D. Finn et al.   BLOOD

Platelet-directed gene therapy overcomes inhibitory antibodies to factor VIII

(2012) M. K. CHUAH et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Lentivirus-mediated platelet gene therapy of murine hemophilia A with pre-existing anti-factor VIII immunity

(2012) E. L. KUETHER et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Chromosomal context and epigenetic mechanisms control the efficacy of genome editing by rare-cutting designer endonucleases

(2012) Fayza Daboussi et al.   NUCLEIC ACIDS RESEARCH

Platelet and endothelial expression of clotting factors for the treatment of hemophilia

(2012) Robert R. Montgomery et al.   THROMBOSIS RESEARCH

Hepatocyte-targeted expression by integrase-defective lentiviral vectors induces antigen-specific tolerance in mice with low genotoxic risk

(2011) Janka Mátrai et al.   HEPATOLOGY

Clinical Progress in Gene Therapy: Sustained Partial Correction of the Bleeding Disorder in Patients Suffering from Severe Hemophilia B

(2011) T. VandenDriessche et al.   HUMAN GENE THERAPY

Gene therapy for haemophilia: a long and winding road

(2011) K. A. HIGH   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

A MicroRNA-regulated and GP64-pseudotyped Lentiviral Vector Mediates Stable Expression of FVIII in a Murine Model of Hemophilia A

(2011) Hideto Matsui et al.   MOLECULAR THERAPY

In vivo genome editing restores haemostasis in a mouse model of haemophilia

(2011) Hojun Li et al.   NATURE

Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B

(2011) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

Platelet gene therapy improves hemostatic function for integrin  IIb 3-deficient dogs

(2011) J. Fang et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Costs of Care in Hemophilia and Possible Implications of Health Care Reform

(2011) K. A. Johnson et al.   Hematology-American Society of Hematology Education Program

Factor IX ectopically expressed in platelets can be stored in  -granules and corrects the phenotype of hemophilia B mice

(2010) G. Zhang et al.   BLOOD

Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B

(2010) V. R. Arruda et al.   BLOOD

Assessing the potential for AAV vector genotoxicity in a murine model

(2010) H. Li et al.   BLOOD

Eradication of neutralizing antibodies to factor VIII in canine hemophilia A after liver gene therapy

(2010) J. D. Finn et al.   BLOOD

Codon optimization of human factor VIII cDNAs leads to high-level expression

(2010) N. J. Ward et al.   BLOOD

Preclinical and clinical progress in hemophilia gene therapy

(2010) Janka Mátrai et al.   CURRENT OPINION IN HEMATOLOGY

Functional aspects of factor VIII expression after transplantation of genetically-modified hematopoietic stem cells for hemophilia A

(2010) Lucienne M. Ide et al.   JOURNAL OF GENE MEDICINE

Recent Advances in Lentiviral Vector Development and Applications

(2010) Janka Mátrai et al.   MOLECULAR THERAPY

Lentiviral Vector Platform for Production of Bioengineered Recombinant Coagulation Factor VIII

(2010) H Trent Spencer et al.   MOLECULAR THERAPY

Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

(2010) Marina Cavazzana-Calvo et al.   NATURE

Specific gene transfer to neurons, endothelial cells and hematopoietic progenitors with lentiviral vectors

(2010) Brigitte Anliker et al.   NATURE METHODS

Correction of murine hemophilia A following nonmyeloablative transplantation of hematopoietic stem cells engineered to encode an enhanced human factor VIII variant using a safety-augmented retroviral vector

(2009) A. Ramezani et al.   BLOOD

A murine model for induction of long-term immunologic tolerance to factor VIII does not require persistent detectable levels of plasma factor VIII and involves contributions from Foxp3+ T regulatory cells

(2009) H. Matsui et al.   BLOOD

In vivo delivery of a microRNA-regulated transgene induces antigen-specific regulatory T cells and promotes immunologic tolerance

(2009) A. Annoni et al.   BLOOD

Emerging potential of transposons for gene therapy and generation of induced pluripotent stem cells

(2009) T. VandenDriessche et al.   BLOOD

The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy

(2009) Eugenio Montini et al.   JOURNAL OF CLINICAL INVESTIGATION

Image-guided, Lobe-specific Hydrodynamic Gene Delivery to Swine Liver

(2009) Kenya Kamimura et al.   MOLECULAR THERAPY

Insertional Transformation of Hematopoietic Cells by Self-inactivating Lentiviral and Gammaretroviral Vectors

(2009) Ute Modlich et al.   MOLECULAR THERAPY

Molecular evolution of a novel hyperactive Sleeping Beauty transposase enables robust stable gene transfer in vertebrates

(2009) Lajos Mátés et al.   NATURE GENETICS

Platelet-delivered factor VIII provides limited resistance to anti-factor VIII inhibitors

(2008) J. GEWIRTZ et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Computer-assisted Hydrodynamic Gene Delivery

(2008) Takeshi Suda et al.   MOLECULAR THERAPY

Efficient, Long-term Hepatic Gene Transfer Using Clinically Relevant HDAd Doses by Balloon Occlusion Catheter Delivery in Nonhuman Primates

(2008) Nicola Brunetti-Pierri et al.   MOLECULAR THERAPY

Effect of Gene Therapy on Visual Function in Leber's Congenital Amaurosis

(2008) James W.B. Bainbridge et al.   NEW ENGLAND JOURNAL OF MEDICINE

Next generation of adeno-associated virus 2 vectors: Point mutations in tyrosines lead to high-efficiency transduction at lower doses

(2008) L. Zhong et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

(2007) Zhijian Wu et al.   MOLECULAR THERAPY