Zobrazeno 1 - 9
of 9
pro vyhledávání: '"Brent Fall"'
Autor:
Brent Fall, C Ronald Scott, Michael Mauer, Stuart Shankland, Jeffrey Pippin, Jonathan A Jefferson, Eric Wallace, David Warnock, Behzad Najafian
Publikováno v:
PLoS ONE, Vol 11, Iss 12, p e0168346 (2016)
Chronic kidney disease is a major complication of Fabry disease. Podocytes accumulate globotriaosylceramide inclusions more than other kidney cell types in Fabry patients. Podocyte injury occurs early in age, and is progressive. Since injured podocyt
Externí odkaz:
https://doaj.org/article/4e3b7e42ecd34ce48d2d9afc4fb7aebc
Autor:
Michael Mauer, Brent Fall, C. Ronald Scott, Behzad Najafian, Eric Wallace, Jeffrey W. Pippin, David G. Warnock, Jonathan Ashley Jefferson, Stuart J. Shankland
Publikováno v:
PLoS ONE, Vol 11, Iss 12, p e0168346 (2016)
PLoS ONE
PLoS ONE
Chronic kidney disease is a major complication of Fabry disease. Podocytes accumulate globotriaosylceramide inclusions more than other kidney cell types in Fabry patients. Podocyte injury occurs early in age, and is progressive. Since injured podocyt
Publikováno v:
Molecular therapy : the journal of the American Society of Gene Therapy. 18(1)
To explore whether stable transduction of myogenic stem cells using lentiviral vectors could be of benefit for treating dystrophic muscles, we generated vectors expressing a functional microdystrophin/enhanced green fluorescence protein fusion (micro
Autor:
Brent Fall, En Kimura, Jeffrey S. Chamberlain, Miki Haraguchi, Sheng Li, Jay J. Han, Jennifer J. Ra, Stephen J. Tapscott
Duchenne muscular dystrophy (DMD) is characterized in skeletal muscle by cycles of myofiber necrosis and regeneration leading to loss of muscle fibers and replacement with fibrotic connective and adipose tissue. The ongoing activation and recruitment
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::f4d7ee4f3173952cb022aa0d624e71cc
https://europepmc.org/articles/PMC2574879/
https://europepmc.org/articles/PMC2574879/
Autor:
Jeffrey S. Chamberlain, Leonard Meuse, John A. Faulkner, Brent Fall, Morayma Reyes, En Kimura, Sheng Li, Rainer Ng
Publikováno v:
Human molecular genetics. 15(10)
A promising approach for treating Duchenne muscular dystrophy (DMD) is by autologous cell transplantation of myogenic stem cells transduced with a therapeutic expression cassette. Development of this method has been hampered by a low frequency of cel
Autor:
En Kimura, Jeffrey S. Chamberlain, John C. Angello, Brent Fall, Stephen D. Hauschka, Sheng Li, Robert E. Welikson, Morayma Reyes
Publikováno v:
Gene therapy. 12(14)
Gene therapy for Duchenne muscular dystrophy (DMD) will require sustained expression of therapeutic dystrophins in striated muscles. Lentiviral vectors have a relatively large transgene carrying capacity and can integrate into nondividing cells. We t
Autor:
Michael Mauer, Cecilia Ponchiardi, Jonathan Ashley Jefferson, Behzad Najafian, Ronald Scott, Brent Fall, Stuart J. Shankland, Chester B. Whitley, Stefanie B. Uhrich, Jeffrey W. Pippin
Publikováno v:
Molecular Genetics and Metabolism. 108:S76-S77
Publikováno v:
Molecular Therapy. 9:S92-S93
Top of pageAbstract To study candidate treatments for Duchenne muscular dystrophy, we have generated a series of lentiviral vectors that express various reporter genes and mini-dystrophin cassettes and tested their ability to transduce a variety of c
Autor:
En Kimura, Xin Ye, John A. Faulkner, Steven D. Hauschka, Leonard Meuse, Sheng Li, Brent Fall, Jeffrey S. Chamberlain
Publikováno v:
Molecular Therapy. 9:S93
Duchenne muscular dystrophy (DMD) results from mutations in the largest known gene, dystrophin. Gene therapy for DMD will require methods to systemically deliver a therapeutic dystrophin gene to widely distributed muscles. Recent observations indicat