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

Production of High-Titer Recombinant Adeno-Associated Virus Vectors in the Absence of Helper Adenovirus

1998 • 1,333 citations

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

2000 • 1,022 citations

Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors

1997 • 588 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

Increased apoptosis of Huntington disease lymphoblasts associated with repeat length-dependent mitochondrial depolarization

1999 • 546 citations

Current protocols in human genetics

1995 • 498 citations

Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus

1997 • 473 citations

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

1999 • 416 citations

Adenovirus mediated expression of therapeutic plasma levels of human factor IX in mice

1993 • 345 citations

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

1993 • 305 citations

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

2000 • 174 citations

The Development of Human Gene Therapy

1999 • 164 citations

Prospects for in Utero Human Gene Therapy

1999 • 155 citations

Role of Vector in Activation of T Cell Subsets in Immune Responses against the Secreted Transgene Product Factor IX

2000 • 134 citations

In UteroGene Therapy: Transfer and Long-Term Expression of the Bacterialneo^rGene in Sheep after Direct Injection of Retroviral Vectors into Preimmune Fetuses

1998 • 126 citations

Prevalence and outcome of intracranial haemorrhage in haemophiliacs – a survey of the paediatric group of the German Society of Thrombosis and Haemostasis (GTH)

1999 • 126 citations

Adenoviral-mediated gene transfer to fetal pulmonary epithelia in vitro and in vivo.

1995 • 105 citations

In utero gene transfer into the pulmonary epithelium

1995 • 98 citations

Short-term correction of factor VIII deficiency in a murine model of hemophilia A after delivery of adenovirus murine factor VIII in utero

1999 • 78 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

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

1997 • 75 citations

Gene therapy for hemophilia

2001 • 67 citations

Adenovirus-Mediated Gene Transfer to the Respiratory Tract of Fetal SheepIn Utero

1995 • 67 citations

Regulatory Issues: Prenatal Gene Transfer: Scientific, Medical, and Ethical Issues. A Report of the Recombinant DNA Advisory Committee

2000 • 62 citations

Long-term gene transfer to mouse fetuses with recombinant adenovirus and adeno-associated virus (AAV) vectors

2000 • 61 citations

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

1999 • 56 citations

Regulation of the phosphoenolpyruvate carboxykinase/human factor IX gene introduced into the livers of adult rats by receptor‐mediated gene transfer

1993 • 55 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

In Utero Cardiac Gene Transfer via Intraplacental Delivery of Recombinant Adenovirus

1997 • 46 citations

Intrauterine gene transfer: gestational stage-specific gene delivery in mice

1999 • 41 citations

Fetal gene therapy: Efficacy, toxicity, and immunologic effects of early gestation recombinant adenovirus

1999 • 39 citations

Adenovirus-mediated gene transfer during initial organogenesis in the mammalian embryo is promoter-dependent and tissue-specific

1997 • 34 citations

Adenovirus-Mediated Gene Transfer in the Midgestation Fetal Mouse

1999 • 34 citations

How do some haemophiliacs develop inhibitors?

1998 • 32 citations

Fetal somatic gene therapy

1996 • 19 citations

Prenatal gene therapy: can the technical hurdles be overcome?

1999 • 17 citations