Should gene therapy be used for newborns with hemophilia?

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

2000 • 1,022 citations

Nonviral Transfer of the Gene Encoding Coagulation Factor VIII in Patients with Severe Hemophilia A

2001 • 366 citations

Adeno-Associated Viral Vector-Mediated Gene Transfer of Human Blood Coagulation Factor IX Into Mouse Liver

1998 • 184 citations

Long-term expression of human coagulation factor VIII and correction of hemophilia A after in vivo retroviral gene transfer in factor VIII-deficient mice

1999 • 179 citations

Therapeutic neonatal hepatic gene therapy in mucopolysaccharidosis VII dogs

2002 • 171 citations

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

1996 • 125 citations

Gene Therapy a Suspect in Leukemia-like Disease

2002 • 117 citations

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

2003 • 111 citations

Inhibitor Antibodies to Factor VIII and Factor IX: Management

2000 • 106 citations

Immune tolerance induction in hemophilia patients with inhibitors: costly can be cheaper

2000 • 95 citations

Evaluation of Pathological Manifestations of Disease in Mucopolysaccharidosis VII Mice after Neonatal Hepatic Gene Therapy

2002 • 69 citations

Gene therapy for hemophilia

2001 • 67 citations

Correlation between Factor VIII Genotype and Inhibitor Development in Hemophilia A

2000 • 55 citations

Gene therapy: reality or myth for the global bleeding disorders community?

2002 • 35 citations

The neonatal immune system

1996 • 29 citations

Haemophilia care in the developing world: benchmarking for excellence

2002 • 28 citations

Immunologic tolerance

1997 • 17 citations

Progress in haemophilic care: ethical issues

2002 • 14 citations

Management of hemophilia in developing countries

2001 • 7 citations