Zobrazeno 1 - 10
of 37
pro vyhledávání: '"Leonard Meuse"'
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
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::ea1dce55bd07a86039709140cce3f050
https://doi.org/10.1182/blood-2003-02-0495
https://doi.org/10.1182/blood-2003-02-0495
Publikováno v:
Molecular Therapy. 8(4):654-665
Transposon-based vectors represent promising new tools for chromosomal transgene insertion and establishment of persistent gene expression in vivo. Here, we report the development of helper-independent transposon-transposase (HITT) vectors, which con
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::7d9cfc890ff5829e8293e60c458f033e
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
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::cd5c23fded5b6897e745805a5a16f46f
Autor:
Kazuo Ohashi, Peter Sarnow, Leonard Meuse, Shiliang Shen, Anton P. McCaffrey, Peter J. Lukavsky, Alissa M. Lancaster, Mark A. Kay
Publikováno v:
Molecular Therapy. 5:676-684
Hepatitis C virus (HCV) is an RNA virus infecting 1 in every 40 people worldwide. Development of new therapeutics for treating HCV has been hampered by the lack of small-animal models. We have adapted existing hydrodynamic transfection methods to opt
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::1c1ffdf7e2b7facd3be4f17a9a90bb5a
https://doi.org/10.1006/mthe.2002.0600
https://doi.org/10.1006/mthe.2002.0600
Publikováno v:
Molecular Therapy. 3:403-410
The short duration of transgene expression remains a major obstacle for the implementation of nonviral DNA vectors in clinical gene therapy trials. Here, we demonstrate stable, long-term transgene expression in vivo by transfecting a linear DNA expre
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::de49fe05aa0ba884a42eb896ae7fd1dd
https://doi.org/10.1006/mthe.2001.0278
https://doi.org/10.1006/mthe.2001.0278
Autor:
Mark A. Kay, Arthur R. Thompson, Xin Ye, Kazuo Ohashi, Gijsbert A. Patijn, Leonard Meuse, Carol H. Miao
Publikováno v:
Molecular Therapy. 1:522-532
We systematically compared human factor IX gene expression from a variety of plasmids containing different cis-regulatory sequences after transfection into different hepatocyte cell lines, or in vivo, after their injection into the livers of mice. Al
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::552e66a4db00f319053bc706167cee20
https://doi.org/10.1006/mthe.2000.0075
https://doi.org/10.1006/mthe.2000.0075
Autor:
Ralph H. Schwall, Harry B. Greenberg, Jeffrey S. Glenn, Bruno B. Bordier, Patricia L. Marion, John M. Cullen, Kazuo Ohashi, Leonard Meuse, Hiroyuki Nakai, Mark A. Kay
Publikováno v:
Nature Medicine. 6:327-331
Persistence of hepatocytes transplanted into the same or related species has been established. The long-term engraftment of human hepatocytes into rodents would be useful for the study of human viral hepatitis, where it might allow the species, techn
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::5f4bb227d5aeadcda3449a3ab30a2b5a
https://doi.org/10.1038/73187
https://doi.org/10.1038/73187
Autor:
Carol H. Miao, Leonard Meuse, Timothy C. Nichols, Brian A. Donahue, Kenneth M. Brinkhous, Richard O. Snyder, Julie Tubb, Marjorie S. Read, Dwight A. Bellinger, Arthur R. Thompson, Mark A. Kay, Salil Patel, Darrel W. Stafford, Hui Feng Lin
Publikováno v:
Nature Medicine. 5:64-70
Hemophilia B, or factor IX deficiency, is an X-linked recessive disorder occurring in about 1 in 25,000 males. Affected individuals are at risk for spontaneous bleeding into many organs; treatment mainly consists of the transfusion of clotting factor
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::aa0f5d600b6cf5228b66090bd522a0b7
https://doi.org/10.1038/4751
https://doi.org/10.1038/4751
Publikováno v:
Human Gene Therapy. 9:1449-1456
Recombinant retroviral vectors represent an attractive means of transferring genes into the liver because they integrate in the host cell genome and result in permanent gene expression. However, efficient gene transfer is limited by the requirement o
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::830680f8d496aa495348f846183ef2ab
https://doi.org/10.1089/hum.1998.9.10-1449
https://doi.org/10.1089/hum.1998.9.10-1449
Autor:
Jeffrey S. Chamberlain, Paul Gregorevic, James M. Allen, Miki Haraguchi, Elina Minami, Eric E. Finn, Michael J. Blankinship, Marvin E. Adams, Leonard Meuse, Stanley C. Froehner, Charles E. Murry
Publikováno v:
Nature Medicine. 12:787-789
Mice carrying mutations in both the dystrophin and utrophin genes die prematurely as a consequence of severe muscular dystrophy. Here, we show that intravascular administration of recombinant adeno-associated viral (rAAV) vectors carrying a microdyst
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::05b74f82f6b8d6af9e37af809b1f264d
https://doi.org/10.1038/nm1439
https://doi.org/10.1038/nm1439