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

Molecular Cloning: A Laboratory Manual

2001 • 133,517 citations

Molecular cloning: A laboratory manual

1990 • 85,659 citations

RAG-1-deficient mice have no mature B and T lymphocytes

1992 • 2,859 citations

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

1996 • 902 citations

Complete nucleotide sequences of the gene for human factor IX (antihemophilic factor B)

1985 • 683 citations

Recombinant adeno-associated virus for muscle directed gene therapy

1997 • 656 citations

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

1996 • 608 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

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

1998 • 489 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

Isolation and characterization of a cDNA coding for human factor IX.

1982 • 415 citations

Gene therapy via primary myoblasts: long-term expression of factor IX protein following transplantation in vivo.

1992 • 313 citations

Reactivation of the Previously Silenced Cytomegalovirus Major Immediate-Early Promoter in the Mouse Liver: Involvement of NFκB

1998 • 283 citations

Efficient and Stable Adeno-Associated Virus-Mediated Transduction in the Skeletal Muscle of Adult Immunocompetent Mice

1997 • 211 citations

Poly(ADP-Ribose) Polymerase Binds with Transcription Enhancer Factor 1 to MCAT1 Elements To Regulate Muscle-Specific Transcription

1999 • 186 citations

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

1998 • 184 citations

A conserved CATTCCT motif is required for skeletal muscle-specific activity of the cardiac troponin T gene promoter.

1988 • 183 citations

Quantitative analysis of the human alpha-skeletal actin gene in transgenic mice.

1993 • 169 citations

Transforming growth factor-beta response elements of the skeletal alpha-actin gene. Combinatorial action of serum response factor, YY1, and the SV40 enhancer-binding protein, TEF-1

1994 • 167 citations

Multiple 5'-flanking regions of the human alpha-skeletal actin gene synergistically modulate muscle-specific expression.

1987 • 150 citations

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

1996 • 125 citations

Long-term correction of obesity and diabetes in genetically obese mice by a single intramuscular injection of recombinant adeno-associated virus encoding mouse leptin

1997 • 124 citations

Optimization of Plasmid Vectors for High-Level Expression in Lung Epithelial Cells

1997 • 116 citations

New developments in the generation of Ad-free, high-titer rAAV gene therapy vectors

1997 • 112 citations

Analysis of the upstream regions governing expression of the chicken cardiac troponin T gene in embryonic cardiac and skeletal muscle cells.

1988 • 109 citations

Analysis of Muscle Creatine Kinase Gene Regulatory Elements in Skeletal and Cardiac Muscles of Transgenic Mice

1996 • 96 citations

Primary myoblast-mediated gene transfer: persistent expression of human factor IX in mice.

1994 • 88 citations

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

1995 • 86 citations

Nucleotide sequence and expression of the human skeletal ?-actin gene: Evolution of functional regulatory domains*1

1988 • 84 citations

Can haemophilic arthropathy be prevented?

1998 • 55 citations

The myosin light chain enhancer and the skeletal actin promoter share a binding site for factors involved in muscle-specific gene expression.

1991 • 42 citations

Prophylactic treatment in Sweden — overtreatment or optimal model?

1998 • 34 citations

Adeno-Associated Virus-Mediated Gene Transfer of Factor IX for Treatment of Hemophilia B by Gene Therapy

1999 • 33 citations

Construction of Human Factor IX Expression Vectors in Retroviral Vector Frames Optimized for Muscle Cells

1996 • 32 citations

Improved adenoviral vectors for gene therapy of Duchenne muscular dystrophy

1997 • 29 citations

Myosin regulatory elements as vectors for gene transfer by intramuscular injection

1998 • 25 citations