M
Melissa A. Rosenfeld
Researcher at National Institutes of Health
Publications - 26
Citations - 6955
Melissa A. Rosenfeld is an academic researcher from National Institutes of Health. The author has contributed to research in topics: Cystic fibrosis & Genetic enhancement. The author has an hindex of 20, co-authored 26 publications receiving 6800 citations. Previous affiliations of Melissa A. Rosenfeld include Cornell University.
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Journal ArticleDOI
Niemann-Pick C1 disease gene: homology to mediators of cholesterol homeostasis.
Eugene D. Carstea,Jill A. Morris,Katherine G. Coleman,Stacie K. Loftus,Dana Zhang,Christiano Cummings,Jessie Z. Gu,Melissa A. Rosenfeld,William J. Pavan,David B. Krizman,James W. Nagle,M H Polymeropoulos,Stephen L. Sturley,Yiannis A. Ioannou,Maureen E. Higgins,Marcella E. Comly,Adele Cooney,Anthony Brown,Christine R. Kaneski,E. Joan Blanchette-Mackie,Nancy K. Dwyer,Edward B. Neufeld,Ta-Yuan Chang,Laura Liscum,Jerome F. Strauss,Kousaku Ohno,Marsha Zeigler,Rivka Carmi,J Sokol,David Markie,Raymond R. O'Neill,O. P. van Diggelen,Milan Elleder,Marc C. Patterson,Roscoe O. Brady,Marie T. Vanier,Peter G. Pentchev,Danilo A. Tagle +37 more
TL;DR: Transfection of NP-C fibroblasts with wild-type NPC1 cDNA resulted in correction of their excessive lysosomal storage of LDL cholesterol, thereby defining the critical role of NPC1 in regulation of intracellular cholesterol trafficking.
Journal ArticleDOI
In vivo transfer of the human cystic fibrosis transmembrane conductance regulator gene to the airway epithelium
Melissa A. Rosenfeld,Kunihiko Yoshimura,Bruce C. Trapnell,Koichi Yoneyama,Eugene Rosenthal,Wilfried Dalemans,Masashi Fukayama,Joachim Bargon,L Stier,Leslie Stratford-Perricaudet,Michel Perricaudet,William B. Guggino,Andrea Pavirani,J P Lecocq,Ronald G. Crystal +14 more
TL;DR: Observations suggest the feasibility of in vivo CFTR gene transfer as therapy for the pulmonary manifestations of CF.
Journal ArticleDOI
Adenovirus-mediated transfer of a recombinant alpha 1-antitrypsin gene to the lung epithelium in vivo
Melissa A. Rosenfeld,Wolfgang Siegfried,Kunihiko Yoshimura,Koichi Yoneyama,Masashi Fukayama,L Stier,Paavo K. Paakko,Pascale Gilardi,L D Stratford-Perricaudet,Michel Perricaudet,Sophie Jallat,Andrea Pavirani,Jean-Pierre Lecocq,Ronald G. Crystal +13 more
TL;DR: 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 by infecting epithelial cells of the cotton rat respiratory tract in vitro and in vivo.
Journal ArticleDOI
Administration of an adenovirus containing the human CFTR cDNA to the respiratory tract of individuals with cystic fibrosis.
Ronald G. Crystal,Noel G. McElvaney,Noel G. McElvaney,Melissa A. Rosenfeld,Chin-Shyan Chu,Andrea Mastrangeli,Andrea Mastrangeli,J. G. Hay,J. G. Hay,Steven L. Brody,H A Jaffe,N. T. Eissa,Claire Danel +12 more
TL;DR: It is feasible to use an adenovirus vector to transfer and express the CFTR cDNA in the respiratory epithelium of individuals with CF and Correction of the CF phenotype of the airway epithelia might be achieved with this strategy.
Journal ArticleDOI
Murine model of Niemann-Pick C disease: mutation in a cholesterol homeostasis gene.
Stacie K. Loftus,Jill A. Morris,Eugene D. Carstea,Jessie Z. Gu,Christiano Cummings,Anthony Brown,Jane S. Ellison,Kousaku Ohno,Melissa A. Rosenfeld,Danilo A. Tagle,Peter G. Pentchev,William J. Pavan +11 more
TL;DR: The mouse model may provide an important resource for studying the role of NPC1 in cholesterol homeostasis and neurodegeneration and for assessing the efficacy of new drugs for NP-C disease.