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

Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain

1994 • 1,032 citations

Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector

1996 • 902 citations

Second-strand synthesis is a rate-limiting step for efficient transduction by recombinant adeno-associated virus vectors

1996 • 747 citations

Helper-free stocks of recombinant adeno-associated viruses: normal integration does not require viral gene expression

1989 • 741 citations

Recombinant adeno-associated virus for muscle directed gene therapy

1997 • 656 citations

MHC class I-cestricted cytotoxic T lymphocytes to viral antigens destroy hepatocytes in mice infected with E1-deleted recombinant adenoviruses

1994 • 619 citations

Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein

1996 • 608 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

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

1997 • 588 citations

Transduction with recombinant adeno-associated virus for gene therapy is limited by leading-strand synthesis

1996 • 542 citations

Stable in vivo expression of the cystic fibrosis transmembrane conductance regulator with an adeno-associated virus vector.

1993 • 485 citations

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

1997 • 473 citations

Immune responses to viral antigens versus transgene product in the elimination of recombinant adenovirus-infected hepatocytes in vivo.

1996 • 383 citations

The Liver: Biology and Pathology

1989 • 348 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

Isolation and Characterization of the Human Chromosomal Gene for Polypeptide Chain Elongation Factor-1α

1989 • 277 citations

Adeno-associated virus vectors preferentially transduce cells in S phase.

1994 • 272 citations

In vivo hepatic gene therapy: complete albeit transient correction of factor IX deficiency in hemophilia B dogs.

1994 • 255 citations

Expression of human alpha 1-antitrypsin in dogs after autologous transplantation of retroviral transduced hepatocytes.

1992 • 248 citations

Persistent expression of human clotting factor IX from mouse liver after intravenous injection of adeno-associated virus vectors

1997 • 245 citations

DNA-damaging agents greatly increase the transduction of nondividing cells by adeno-associated virus vectors

1994 • 227 citations

DNA synthesis and topoisomerase inhibitors increase transduction by adeno-associated virus vectors.

1995 • 220 citations

Effect of the E4 region on the persistence of transgene expression from adenovirus vectors

1997 • 195 citations

Hepatic Gene Therapy: Persistent Expression of Human α1-Antitrypsin in Mice after Direct Gene Delivery In Vivo

1992 • 178 citations

Production of human factor IX in animals by genetically modified skin fibroblasts: potential therapy for hemophilia B

1989 • 177 citations

The enhancer domain of the human cytomegalovirus major immediate-early promoter determines cell type-specific expression in transgenic mice

1996 • 169 citations

Evaluation of promoter strength for hepatic gene expression in vivo following adenovirus-mediated gene transfer.

1996 • 143 citations

Adeno-associated virus vectors transduce primary cells much less efficiently than immortalized cells

1995 • 139 citations

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

1996 • 125 citations

Molecular Basis of Thrombosis and Hemostasis

1996 • 119 citations

Expression of human factor IX in rat capillary endothelial cells: toward somatic gene therapy for hemophilia B.

1991 • 116 citations

Human hepatoma cells secrete single chain factor X, prothrombin, and antithrombin III

1984 • 97 citations

A Novel Adenovirus—Adeno-Associated Virus Hybrid Vector That Displays Efficient Rescue and Delivery of the AAV Genome

1996 • 92 citations

Role of Intron I in Expression of the Human Factor IX Gene

1995 • 86 citations

Persistent Systemic Production of Human Factor IX in Mice by Skeletal Myoblast-Mediated Gene Transfer: Feasibility of Repeat Application to Obtain Therapeutic Levels

1997 • 44 citations

Current therapeutic relevance of liver gene transfer Kay MA, Rothenberg S, Landen CN, Bellinger DA, Leland F, Toman C, Finegold M, Thompson AR, Read MS, Brinkhous KM, Woo SLC. In vivo gene therapy of hemophilia B: sustained partial correction on factor IX-deficient dogs. Science 1993;262:117?119

1994 • 35 citations

The A2L intermediate gene product is required for in vitro transcription from a vaccinia virus late promoter

1996 • 19 citations

AAV vectors can be efficiently produced without helper virus

1995 • 12 citations

[1] Adeno-associated virus: Integrating vectors for human gene therapy

1995 • 6 citations

A New Form of Strange Matter And New Hope for Finding It

1993 • 2 citations