Zobrazeno 1 - 10
of 18
pro vyhledávání: '"Sally Fuess"'
Autor:
Helen M. Blau, Christian Trautwein, D. Denison Jenkins, Joseph Lin, Judith A. Shizuru, Regis Doyonnas, Scott V. Perryman, Karl G. Sylvester, Konrad L. Streetz, Dirk Grimm, Mark A. Kay, Sally Fuess
Publikováno v:
Hepatology. 47:706-718
The lack of adequate donor organs is a major limitation to the successful widespread use of liver transplantation for numerous human hepatic diseases. A desirable alternative therapeutic option is hepatocyte transplantation (HT), but this approach is
Autor:
Bassel Akache, Dariya S. Glazer, Julie Park, Mark A. Kay, Sally Fuess, Dirk Grimm, Xuan Shen, Stephen R. Yant
Publikováno v:
Molecular Therapy
Vectors based on different serotypes of adeno-associated virus hold great promise for human gene therapy, based on their unique tissue tropisms and distinct immunological profiles. A particularly interesting candidate is AAV8, which can efficiently a
Autor:
Katsuya Inagaki, Gregory A. Gibson, Theresa A. Storm, Mark A. Kay, Sally Fuess, Charles F. McTiernan, Hiroyuki Nakai
Publikováno v:
Molecular Therapy. 14(1):45-53
It has been recently shown that recombinant adeno-associated virus serotype 8 (rAAV8) is a robust alternative serotype vector that overcomes many of the limitations of rAAV2 and transduces various tissues efficiently and globally through systemic vec
Autor:
Hiroyuki Nakai, Michael A. Lochrie, Shangzhen Zhou, Takashi Matsushita, Alan McClelland, Leonard Meuse, Richard T. Surosky, Mark A. Kay, Sally Fuess, Dirk Grimm, Clare E. Thomas, James R. Allen, Peter Colosi, Theresa A. Storm
Publikováno v:
Blood. 102:2412-2419
We report the generation and use of pseudotyped adeno-associated viral (AAV) vectors for the liver-specific expression of human blood coagulation factor IX (hFIX). Therefore, an AAV-2 genome encoding the hfIX gene was cross-packaged into capsids of A
Publikováno v:
Human Gene Therapy. 14:871-881
Elucidation of the mechanisms of transformation of single-stranded (ss) recombinant adeno-associated virus (rAAV) vector genomes into a variety of stable double-stranded (ds) forms is key to a complete understanding of rAAV vector transduction in viv
Autor:
Leonard Meuse, Mark A. Kay, Sally Fuess, Theresa A. Storm, Milton J. Finegold, Markus Grompe, Hiroyuki Nakai, Eugenio Montini
Publikováno v:
Molecular Therapy. 7(1):101-111
Nonviral plasmid DNA is a promising vector for achieving ex vivo and in vivo gene transfer. However, transgene expression is usually transient, especially in dividing target cells due to loss of vector genomes. Here we describe the use of naked doubl
Autor:
Susanna M. Lewis, Theresa A. Storm, David J. Munroe, Hiroyuki Nakai, Congrong Ma, Katsuya Inagaki, Xiaolin Wu, Mark A. Kay, Sally Fuess
Publikováno v:
Journal of virology. 81(20)
Adeno-associated virus (AAV) is a small nonpathogenic, replication-defective DNA virus with a single-stranded DNA genome. Recombinant AAV (rAAV) is among the most promising gene delivery vectors for human gene therapy (15, 28). rAAV lacks machinery f
Publikováno v:
Molecular Therapy. 13
rAAV8 vectors transduce various tissues with extremely high efficiency in neonates and infants. Many applications of rAAV8 vector injection into prenatal or neonatal mice have been proposed to treat various disorders. Intraperitoneal or intravascular
Publikováno v:
Journal of virology. 79(1)
Recombinant adeno-associated virus (rAAV) vectors can mediate long-term stable transduction in various target tissues. However, with rAAV serotype 2 (rAAV2) vectors, liver transduction is confined to only a small portion of hepatocytes even after adm
Publikováno v:
Nature genetics. 34(3)
Recombinant adeno-associated virus serotype 2 (rAAV2) is a promising vector for gene therapy because it can achieve long-term stable transgene expression in animals and human subjects after direct administration of vectors into various target tissues