The gene therapy journey for hemophilia: are we there yet?

Activated factor VII: my story

(2012) U. HEDNER   HAEMOPHILIA

Pharmacological Modulation of Humoral Immunity in a Nonhuman Primate Model of AAV Gene Transfer for Hemophilia B

(2012) Federico Mingozzi et al.   MOLECULAR THERAPY

Prolonged half-life and preserved enzymatic properties of factor IX selectively PEGylated on native N-glycans in the activation peptide

(2011) H. Ostergaard et al.   BLOOD

Recombinant factor IX-Fc fusion protein (rFIXFc) demonstrates safety and prolonged activity in a phase 1/2a study in hemophilia B patients

(2011) A. D. Shapiro et al.   BLOOD

Adeno-Associated Virus Antibody Profiles in Newborns, Children, and Adolescents

(2011) Roberto Calcedo et al.   Clinical and Vaccine Immunology

Good Manufacturing Practice Production of Self-Complementary Serotype 8 Adeno-Associated Viral Vector for a Hemophilia B Clinical Trial

(2011) James A. Allay et al.   HUMAN GENE THERAPY

A 10 Patient Case Report on the Impact of Plasmapheresis Upon Neutralizing Factors Against Adeno-associated Virus (AAV) Types 1, 2, 6, and 8

(2011) Virginie Monteilhet et al.   MOLECULAR THERAPY

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

Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges

(2011) Federico Mingozzi et al.   NATURE REVIEWS GENETICS

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

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

Alternative Strategies for Gene Therapy of Hemophilia

(2011) R. R. Montgomery et al.   Hematology-American Society of Hematology Education Program

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

In vivo efficacy of platelet-delivered, high specific activity factor VIII variants

(2010) T. K. Greene et al.   BLOOD

Assessing the potential for AAV vector genotoxicity in a murine model

(2010) H. Li et al.   BLOOD

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

(2010) N. J. Ward et al.   BLOOD

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

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

Worldwide Epidemiology of Neutralizing Antibodies to Adeno‐Associated Viruses

(2009) Roberto Calcedo et al.   JOURNAL OF INFECTIOUS DISEASES

Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial

(2009) Albert M Maguire et al.   LANCET

Host and Vector-dependent Effects on the Risk of Germline Transmission of AAV Vectors

(2009) Patricia Favaro 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

Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency

(2009) Alessandro Aiuti et al.   NEW ENGLAND JOURNAL OF MEDICINE

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

(2008) J. Sun et al.   BLOOD

Manufacturing and characterizing AAV-based vectors for use in clinical studies

(2008) J F Wright   GENE THERAPY

Treatment of Leber Congenital Amaurosis Due to RPE65 Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results of a Phase I Trial

(2008) William W. Hauswirth et al.   HUMAN GENE THERAPY

Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis

(2008) Albert M. Maguire et al.   NEW ENGLAND JOURNAL OF MEDICINE

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

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