Preclinical in vivo evaluation of pseudotyped adeno-associated virus vectors for liver gene therapy

Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy

2002 • 1,463 citations

Membrane-Associated Heparan Sulfate Proteoglycan Is a Receptor for Adeno-Associated Virus Type 2 Virions

1998 • 1,369 citations

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

2000 • 1,022 citations

Cross-Packaging of a Single Adeno-Associated Virus (AAV) Type 2 Vector Genome into Multiple AAV Serotypes Enables Transduction with Broad Specificity

2002 • 737 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

Gene Therapy Vectors Based on Adeno-Associated Virus Type 1

1999 • 529 citations

Adeno-associated virus vectors can be efficiently produced without helper virus

1998 • 489 citations

Infectious Clones and Vectors Derived from Adeno-Associated Virus (AAV) Serotypes Other Than AAV Type 2

1998 • 450 citations

Adeno-Associated Virus Serotype 4 (AAV4) and AAV5 Both Require Sialic Acid Binding for Hemagglutination and Efficient Transduction but Differ in Sialic Acid Linkage Specificity

2001 • 422 citations

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

1999 • 416 citations

Helper virus-free, optically controllable, and two-plasmid-based production of adeno-associated virus vectors of serotypes 1 to 6

2003 • 335 citations

Cloning and Characterization of Adeno-Associated Virus Type 5

1999 • 332 citations

Genetics of adeno-associated virus: isolation and preliminary characterization of adeno-associated virus type 2 mutants

1984 • 303 citations

Inclusion of the Hepatic Locus Control Region, an Intron, and Untranslated Region Increases and Stabilizes Hepatic Factor IX Gene Expression in Vivo but Not in Vitro

2000 • 241 citations

Cloning of adeno-associated virus type 4 (AAV4) and generation of recombinant AAV4 particles

1997 • 230 citations

Hybrid Vectors Based on Adeno-Associated Virus Serotypes 2 and 5 for Muscle-Directed Gene Transfer

2001 • 209 citations

Isolation of Recombinant Adeno-Associated Virus Vector-Cellular DNA Junctions from Mouse Liver

1999 • 184 citations

Nucleotide Sequencing and Generation of an Infectious Clone of Adeno-Associated Virus 3

1996 • 161 citations

Noninvasive gene transfer to the lung for systemic delivery of therapeutic proteins

2002 • 135 citations

Nonrandom Transduction of Recombinant Adeno-Associated Virus Vectors in Mouse Hepatocytes In Vivo: Cell Cycling Does Not Influence Hepatocyte Transduction

2000 • 127 citations

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

1996 • 125 citations

Human Adeno-Associated Virus Type 5 Is Only Distantly Related to Other Known Primate Helper-Dependent Parvoviruses

1999 • 122 citations

Progress in Adeno-Associated Virus Type 2 Vector Production: Promises and Prospects for Clinical Use

1999 • 120 citations

Noninvasive gene transfer to the lung for systemic delivery of therapeutic proteins

2002 • 104 citations

Gene therapy for the hemophilias

1999 • 94 citations

Secreted and Transmembrane Mucins Inhibit Gene Transfer with AAV4 More Efficiently than AAV5

2002 • 86 citations

Improved Hepatic Gene Transfer by Using an Adeno-Associated Virus Serotype 5 Vector

2002 • 72 citations

Viral vectors : basic science and gene therapy

2000 • 31 citations

Adeno-Associated Virus —Mediated Gene Transfer for Hemophilia B

2002 • 18 citations