In utero gene transfer of human factor IX to fetal mice can induce postnatal tolerance of the exogenous clotting factor

‘Actively Acquired Tolerance’ of Foreign Cells

1953 • 3,103 citations

Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector

2000 • 1,022 citations

Cellular and humoral immune responses to adenoviral vectors containing factor IX gene: tolerization of factor IX and vector antigens allows for long-term expression.

1995 • 599 citations

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

1999 • 556 citations

Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors

1999 • 416 citations

Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy

2002 • 336 citations

Evaluation of the Transfer and Expression in Mice of an Enzyme-Encoding Gene Using a Human Adenovirus Vector

1990 • 325 citations

Adenovirus-mediated gene transfer: influence of transgene, mouse strain and type of immune response on persistence of transgene expression

1997 • 186 citations

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

1997 • 163 citations

In Utero Delivery of Adeno-Associated Viral Vectors: Intraperitoneal Gene Transfer Produces Long-Term Expression

2001 • 161 citations

Influence of Vector Dose on Factor IX-Specific T and B Cell Responses in Muscle-Directed Gene Therapy

2002 • 143 citations

Gene Therapy for Hemophilia B: Host Immunosuppression Prolongs the Therapeutic Effect of Adenovirus-Mediated Factor IX Expression

1995 • 127 citations

Successful expression of human factor IX following repeat administration of adenoviral vector in mice.

1996 • 125 citations

Stable delivery of physiologic levels of recombinant erythropoietin to the systemic circulation by intramuscular injection of replication-defective adenovirus.

1994 • 121 citations

Inhibitor Antibodies to Factor VIII and Factor IX: Management

2000 • 106 citations

Persistent expression of canine factor IX in hemophilia B canines

1999 • 105 citations

Successful expression of β-galactosidase and factor IX transgenes in fetal and neonatal sheep after ultrasound-guided percutaneous adenovirus vector administration into the umbilical vein

1999 • 76 citations

Improved muscle-derived expression of human coagulation factor IX from a skeletal actin/CMV hybrid enhancer/promoter

2000 • 75 citations

Foetal gene delivery in mice by intra-amniotic administration of retroviral producer cells and adenovirus

1997 • 75 citations

Sustained delivery of therapeutic concentrations of human clotting factor IX – a comparison of adenoviral and AAV vectors administered in utero

2002 • 63 citations

Improved muscle-derived expression of human coagulation factor IX from a skeletal actin/CMV hybrid enhancer/promoter

2000 • 62 citations

Reexpression Following Readministration of an Adenoviral Vector in Adult Mice after Initial in Utero Adenoviral Administration

2000 • 58 citations

Temporally regulated expression patterns following in utero adenovirus-mediated gene transfer

1999 • 56 citations

Intravenous administration of an E1/E3-deleted adenoviral vector induces tolerance to factor IX in C57BL/6 mice

2001 • 48 citations

Therapeutic plasma concentrations of human factor IX in mice after gene delivery into the amniotic cavity: a model for the prenatal treatment of haemophilia B

1999 • 46 citations

Pharmaco‐economic aspects of inhibitor treatment

1998 • 38 citations

Factor IX Antibody and Immune Tolerance

1999 • 15 citations