Zobrazeno 1 - 10
of 15
pro vyhledávání: '"Melissa A. Rosenfeld"'
Autor:
Rivka Carmi, Laura Liscum, Marcella E. Comly, Christine R. Kaneski, Marsha Zeigler, Adele Cooney, Roscoe O. Brady, Yiannis A. Ioannou, Peter G. Pentchev, Marc C. Patterson, James W. Nagle, E. Joan Blanchette-Mackie, Maureen E. Higgins, David B. Krizman, J Sokol, Raymond R. O'Neill, Jill A. Morris, Jerome F. Strauss, Stephen L. Sturley, Christiano Cummings, O. P. van Diggelen, Ta-Yuan Chang, Edward B. Neufeld, Danilo A. Tagle, Katherine G. Coleman, Jessie Z. Gu, Kousaku Ohno, Stacie K. Loftus, M H Polymeropoulos, Nancy K. Dwyer, David Markie, Melissa A. Rosenfeld, Marie T. Vanier, Anthony Brown, Eugene D. Carstea, Milan Elleder, Dana Zhang, William J. Pavan
Publikováno v:
Science, 277, 228-231. American Association for the Advancement of Science
Niemann-Pick type C (NP-C) disease, a fatal neurovisceral disorder, is characterized by lysosomal accumulation of low density lipoprotein (LDL)–derived cholesterol. By positional cloning methods, a gene (NPC1)with insertion, deletion, and missense
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::5537b8141c320370b8c9c9951d5ad1a0
https://doi.org/10.1126/science.277.5323.228
https://doi.org/10.1126/science.277.5323.228
Autor:
Melissa A. Rosenfeld, Jill A. Morris, Danilo A. Tagle, Jessie Z. Gu, Stacie K. Loftus, Christiano Cummings, Eugene D. Carstea, Peter G. Pentchev, Anthony Brown, Kousaku Ohno, Jane S. Ellison, William J. Pavan
Publikováno v:
Science. 277:232-235
An integrated human-mouse positional candidate approach was used to identify the gene responsible for the phenotypes observed in a mouse model of Niemann-Pick type C (NP-C) disease. The predicted murine NPC1 protein has sequence homology to the putat
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::2f70ec35d179b91e99a914b868c7a197
https://doi.org/10.1126/science.277.5323.232
https://doi.org/10.1126/science.277.5323.232
Publikováno v:
Human Gene Therapy. 5:1121-1129
Cystic fibrosis (CF) results from mutations of the CF transmembrane conductance regulator (CFTR) gene and the consequent defective regulation of cAMP-stimulated Cl- permeability across epithelial cell apical membranes. Given that in vitro transfer of
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::712e4ef31abdb8a004ddbf537933db63
https://doi.org/10.1089/hum.1994.5.9-1121
https://doi.org/10.1089/hum.1994.5.9-1121
Autor:
Chin-Shyan Chu, Claire Danel, Tyrone C. Banks, Ronald G. Crystal, Prem Seth, Kunihiko Yoshimura, Melissa A. Rosenfeld, Koichi Yoneyama
Publikováno v:
Human Gene Therapy. 5:331-342
Cystic fibrosis (CF) results from mutations of the CF transmembrane conductance regulator (CFTR) gene and subsequent defective regulation of cAMP-stimulated chloride (Cl-) permeability across the apical membrane of epithelial cells. In vitro transfer
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::96d1050a79c691ec5556ea02e88d0a07
https://doi.org/10.1089/hum.1994.5.3-331
https://doi.org/10.1089/hum.1994.5.3-331
Publikováno v:
Journal of Biological Chemistry. 268:2300-2303
The present study demonstrates that the human adenovirus (Ad) can augment transfer and expression of a gene within plasmid DNA unmodified by nonspecific linkers or by linker-ligand complexes. Following the transfection of COS-7 cells with pRSVL, a lu
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::ff0ed36b187fcf3904caf6dff5d437e5
https://doi.org/10.1016/s0021-9258(18)53773-x
https://doi.org/10.1016/s0021-9258(18)53773-x
Autor:
Michel Perricaudet, Ronald G. Crystal, J P Lecocq, Melissa A. Rosenfeld, Andrea Pavirani, Andrea Mastrangeli, Claire Danel, L D Stratford-Perricaudet
Publikováno v:
Journal of Clinical Investigation. 91:225-234
A variety of pulmonary disorders, including cystic fibrosis, are potentially amenable to treatment in which a therapeutic gene is directly transferred to the bronchial epithelium. This is difficult to accomplish because the majority of airway epithel
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::14cd4a904c6d548e61ff5c3334bae694
https://doi.org/10.1172/jci116175
https://doi.org/10.1172/jci116175
Autor:
Ronald G. Crystal, Melissa A. Rosenfeld, Hidenori Nakamura, Jean-Pierre Lecocq, Eva M. Scherer, Kunihiko Yoshimura, Andrea Pavirani
Publikováno v:
Nucleic Acids Research. 20:3233-3240
As an approach to gene therapy for the respiratory manifestations of cystic fibrosis (CF), in vivo plasmid-mediated direct transfer of the normal CF transmembrane conductance regulator (CFTR) gene to the airway epithelium was investigated in mice. To
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::bd71bf46e78eb21deea9b73c87d783ca
https://doi.org/10.1093/nar/20.12.3233
https://doi.org/10.1093/nar/20.12.3233
Autor:
L D Stratford-Perricaudet, Koichi Yoneyama, Ronald G. Crystal, L Stier, Paavo K. Paakko, Wolfgang Siegfried, Melissa A. Rosenfeld, Jean-Pierre Lecocq, Sophie Jallat, Michel Perricaudet, Pascale Gilardi, Kunihiko Yoshimura, Masashi Fukayama, Andrea Pavirani
Publikováno v:
Science. 252:431-434
The respiratory epithelium is a potential site for somatic gene therapy for the common hereditary disorders alpha 1-antitrypsin (alpha 1AT) deficiency and cystic fibrosis. A replication-deficient adenoviral vector (Ad-alpha 1AT) containing an adenovi
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::994dfe695648dac65a9583fa671e0e45
https://doi.org/10.1126/science.2017680
https://doi.org/10.1126/science.2017680
Autor:
William B. Guggino, Yuan Jiang, Michael L. Bittner, Karla A. Henning, Elizabeth A. Novotny, Melissa A. Rosenfeld, Peter J. Mogayzel, Erik M. Schwiebert
Publikováno v:
Human molecular genetics. 6(1)
The cystic fibrosis transmembrane conductance regulator gene (CFTR) encodes a transmembrane protein (CFTR) which functions in part as a cyclic adenosine monophosphate (cAMP)-regulated chloride channel. CFTR expression is controlled temporally and cel
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::0886470077477a8a5f565b327212156d
https://pubmed.ncbi.nlm.nih.gov/9002671
https://pubmed.ncbi.nlm.nih.gov/9002671
Autor:
Melissa A. Rosenfeld, Tony Eissa, Claire Danel, Ronald G. Crystal, Ari Jaffe, John G. Hay, Noel G. McElvaney, Chin-Shyan Chu, Steve Brody, Andrea Mastrangeli
Publikováno v:
Human gene therapy. 6(5)
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::624e94644c4289d0c731aef1bead293c
https://pubmed.ncbi.nlm.nih.gov/7578401
https://pubmed.ncbi.nlm.nih.gov/7578401