Haemophilia gene therapy: From trailblazer to gamechanger

Gene Therapy with BMN 270 Results in Therapeutic Levels of FVIII in Mice and Primates and Normalization of Bleeding in Hemophilic Mice

(2018) Stuart Bunting et al.   MOLECULAR THERAPY

Hyperactive Factor IX Padua: A Game-Changer for Hemophilia Gene Therapy

(2018) Thierry VandenDriessche et al.   MOLECULAR THERAPY

Hemophilia Gene Therapy: Ready for Prime Time?

(2017) Thierry VandenDriessche et al.   HUMAN GENE THERAPY

Recombinant Adeno-Associated Viral Integration and Genotoxicity: Insights from Animal Models

(2017) Randy J. Chandler et al.   HUMAN GENE THERAPY

Advances in Gene Therapy for Hemophilia

(2017) Amit C. Nathwani et al.   HUMAN GENE THERAPY

Gene Therapy for Hemophilia

(2017) Arthur W. Nienhuis et al.   MOLECULAR THERAPY

A Novel Platform for Immune Tolerance Induction in Hemophilia A Mice

(2017) Simone Merlin et al.   MOLECULAR THERAPY

A Cornucopia of Therapies under Study for Hemophilia

(2017) Glenn F. Pierce et al.   MOLECULAR THERAPY

Identification of liver-specific enhancer–promoter activity in the 3′ untranslated region of the wild-type AAV2 genome

(2017) Grant J Logan et al.   NATURE GENETICS

Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant

(2017) Lindsey A. George et al.   NEW ENGLAND JOURNAL OF MEDICINE

AAV5–Factor VIII Gene Transfer in Severe Hemophilia A

(2017) Savita Rangarajan et al.   NEW ENGLAND JOURNAL OF MEDICINE

2017 Clinical trials update: Innovations in hemophilia therapy

(2016) Jan Hartmann et al.   AMERICAN JOURNAL OF HEMATOLOGY

Single synonymous mutation in factor IX alters protein properties and underlies haemophilia B

(2016) Vijaya L Simhadri et al.   JOURNAL OF MEDICAL GENETICS

Novel factor VIII variants with a modified furin cleavage site improve the efficacy of gene therapy for hemophilia A

(2016) G. N. Nguyen et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Baboon envelope pseudotyped lentiviral vectors efficiently transduce human B cells and allow active factor IX B cell secretionin vivoin NOD/SCIDγc-/-mice

(2016) C. Levy et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Overexpression of factor VIII after AAV delivery is transiently associated with cellular stress in hemophilia A mice

(2016) Amy M Lange et al.   Molecular Therapy-Methods & Clinical Development

AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice

(2015) J. M. Crudele et al.   BLOOD

In vivo genome editing of the albumin locus as a platform for protein replacement therapy

(2015) R. Sharma et al.   BLOOD

Functional Correction of Large Factor VIII Gene Chromosomal Inversions in Hemophilia A Patient-Derived iPSCs Using CRISPR-Cas9

(2015) Chul-Yong Park et al.   Cell Stem Cell

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

(2015) Paul E. Monahan et al.   HUMAN GENE THERAPY

Adeno-Associated Virus Type 2 and Hepatocellular Carcinoma?

(2015) Kenneth I. Berns et al.   HUMAN GENE THERAPY

Adeno-associated viral vectors for the treatment of hemophilia

(2015) Katherine A. High et al.   HUMAN MOLECULAR GENETICS

Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy

(2015) Randy J. Chandler et al.   JOURNAL OF CLINICAL INVESTIGATION

The important role of von Willebrand factor in platelet-derived FVIII gene therapy for murine hemophilia A in the presence of inhibitory antibodies

(2015) Q. Shi et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Gene therapy in an era of emerging treatment options for hemophilia B

(2015) P. E. Monahan   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Intraosseous Delivery of Lentiviral Vectors Targeting Factor VIII Expression in Platelets Corrects Murine Hemophilia A

(2015) Xuefeng Wang et al.   MOLECULAR THERAPY

Adeno-associated Vector Toxicity—To Be or Not to Be?

(2015) Hildegard Büning et al.   MOLECULAR THERAPY

Recurrent AAV2-related insertional mutagenesis in human hepatocellular carcinomas

(2015) Jean-Charles Nault et al.   NATURE GENETICS

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

(2015) Alessio Cantore et al.   Science Translational Medicine

Omental implantation of BOECs in hemophilia dogs results in circulating FVIII antigen and a complex immune response

(2014) M. C. Ozelo et al.   BLOOD

Computationally designed liver-specific transcriptional modules and hyperactive factor IX improve hepatic gene therapy

(2014) N. Nair et al.   BLOOD

Gene Therapy for Hemophilia: The Clot Thickens

(2014) Katherine A. High   HUMAN GENE THERAPY

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

(2014) Marinee K Chuah et al.   MOLECULAR THERAPY

Hyperactive PiggyBac Transposons for Sustained and Robust Liver-targeted Gene Therapy

(2014) Mario Di Matteo et al.   MOLECULAR THERAPY

Bioengineered coagulation factor VIII enables long-term correction of murine hemophilia A following liver-directed adeno-associated viral vector delivery

(2014) Harrison C Brown et al.   Molecular Therapy-Methods & Clinical Development

Robust ZFN-mediated genome editing in adult hemophilic mice

(2013) X. M. Anguela et al.   BLOOD

Minimal modification in the factor VIII B-domain sequence ameliorates the murine hemophilia A phenotype

(2013) J. I. Siner et al.   BLOOD

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

(2013) A. T. Martino et al.   BLOOD

Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant

(2013) J. McIntosh et al.   BLOOD

Effective gene therapy for haemophilic mice with pathogenic factor IX antibodies

(2013) David M. Markusic et al.   EMBO Molecular Medicine

Liver gene therapy by lentiviral vectors reverses anti-factor IX pre-existing immunity in haemophilic mice

(2013) Andrea Annoni et al.   EMBO Molecular Medicine

Selection and evaluation of clinically relevant AAV variants in a xenograft liver model

(2013) Leszek Lisowski et al.   NATURE

Incorporation of the factor IX Padua mutation into FIX-Triple improves clotting activity in vitro and in vivo

(2013) Chung-Yang Kao et al.   THROMBOSIS AND HAEMOSTASIS

Platelet-targeted gene therapy with human factor VIII establishes haemostasis in dogs with haemophilia A

(2013) Lily M. Du et al.   Nature Communications

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

Recent Progress in Gene Therapy for Hemophilia

(2012) Marinee K. Chuah et al.   HUMAN GENE THERAPY

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

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

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

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

Recent Advances in Lentiviral Vector Development and Applications

(2010) Janka Mátrai et al.   MOLECULAR THERAPY

Efficacy and Safety of Long-term Prophylaxis in Severe Hemophilia A Dogs Following Liver Gene Therapy Using AAV Vectors

(2010) Denise E Sabatino et al.   MOLECULAR THERAPY

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

Capsid antigen presentation flags human hepatocytes for destruction after transduction by adeno-associated viral vectors

(2009) Gary C. Pien et al.   JOURNAL OF CLINICAL INVESTIGATION

Role of regulatory T cells in tolerance to coagulation factors

(2009) O. CAO et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Directed Engineering of a High-expression Chimeric Transgene as a Strategy for Gene Therapy of Hemophilia A

(2009) Christopher B Doering 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

X-Linked Thrombophilia with a Mutant Factor IX (Factor IX Padua)

(2009) Paolo Simioni et al.   NEW ENGLAND JOURNAL OF MEDICINE

In Vitro and In Vivo Gene Therapy Vector Evolution via Multispecies Interbreeding and Retargeting of Adeno-Associated Viruses

(2008) D. Grimm et al.   JOURNAL OF VIROLOGY

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

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