Zobrazeno 1 - 10
of 16
pro vyhledávání: '"Lisa M Petek"'
Autor:
Gregory J Block, Lisa M Petek, Divya Narayanan, Amanda M Amell, James M Moore, Natalia A Rabaia, Ashlee Tyler, Silvere M van der Maarel, Rabi Tawil, Galina N Filippova, Daniel G Miller
Publikováno v:
PLoS ONE, Vol 7, Iss 4, p e35532 (2012)
Facioscapulohumeral Disease (FSHD) is a dominantly inherited progressive myopathy associated with aberrant production of the transcription factor, Double Homeobox Protein 4 (DUX4). The expression of DUX4 depends on an open chromatin conformation of t
Externí odkaz:
https://doaj.org/article/2b50075e83994ad482e31852db218bdd
Autor:
Daniel G. Miller, Leslie Caron, Heather Main, Devaki Kher, Lisa M. Petek, Alejandro Hidalgo, Robert McKernan, Uli Schmidt, Lorenz Poellinger, Giulia Ferri, Kian Leong Lee, Henry Yang, Davide Gabellini, Jia Li, Biljana Dumevska
Publikováno v:
Stem Cells Translational Medicine, Vol 5, Iss 9, Pp 1145-1161 (2016)
Facioscapulohumeral muscular dystrophy (FSHD) represents a major unmet clinical need arising from the progressive weakness and atrophy of skeletal muscles. The dearth of adequate experimental models has severely hampered our understanding of the dise
Autor:
Sandra L. Poliachik, Christopher B. Budech, Dennis W. W. Shaw, Amanda M. Rickard, Lisa M. Petek, Seth D. Friedman, Rabi Tawil, Mark R. Ferguson, Daniel G. Miller
Publikováno v:
Neuromuscular disorders : NMD
Measuring the severity and progression of facioscapulohumeral muscular dystrophy (FSHD) is particularly challenging because muscle weakness progresses over long periods of time and can be sporadic. Biomarkers are essential for measuring disease burde
Publikováno v:
Human Molecular Genetics. 24:5901-5914
Facioscapulohumeral muscular dystrophy (FSHD) is caused by chromatin relaxation that results in aberrant expression of the transcription factor Double Homeobox 4 (DUX4). DUX4 protein is present in a small subset of FSHD muscle cells, making its detec
Autor:
Pei Rong Wang, David W. Russell, Roli K. Hirata, Daniel G. Miller, Mark S. Sands, Lisa M. Petek
Publikováno v:
Nature Biotechnology. 24:1022-1026
Therapeutic gene delivery typically involves the addition of a transgene expression cassette to mutant cells. This approach is complicated by transgene silencing, aberrant transcriptional regulation and insertional mutagenesis. An alternative strateg
Autor:
Lisa M. Petek, David W. Russell, Michael A. Jacobs, Rajinder Kaul, Daniel G. Miller, Grant D. Trobridge
Publikováno v:
Journal of Virology. 79:11434-11442
The integration sites of viral vectors used in human gene therapy can have important consequences for safety and efficacy. However, an extensive evaluation of adeno-associated virus (AAV) vector integration sites has not been completed, despite the o
Publikováno v:
Molecular and Cellular Biology. 23:3550-3557
The use of adeno-associated virus (AAV) to package gene-targeting vectors as single-stranded linear molecules has led to significant improvements in mammalian gene-targeting frequencies. However, the molecular basis for the high targeting frequencies
Autor:
Divya Narayanan, Kathryn C. Davidson, Randall T. Moon, Amanda M. Amell, Thomas D. Bird, Daniel G. Miller, Lisa M. Petek, Rabi Tawil, Gregory J. Block
Facioscapulohumeral muscular dystrophy is a dominantly inherited myopathy associated with chromatin relaxation of the D4Z4 macrosatellite array on chromosome 4. DUX4 is encoded within each unit of the D4Z4 array where it is normally transcriptionally
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::ca490927979e71f341f72a6a052e9d44
https://europepmc.org/articles/PMC3820129/
https://europepmc.org/articles/PMC3820129/
Autor:
Gregory J. Block, Daniel G. Miller, Amanda M. Amell, Rabi Tawil, Natalia A. Rabaia, Galina N. Filippova, Lisa M. Petek, James M. Moore, Divya Narayanan, Ashlee E. Tyler, Silvère M. van der Maarel
Publikováno v:
PLoS ONE, Vol 7, Iss 4, p e35532 (2012)
PLoS ONE
PLoS ONE, 7(4)
PLoS ONE
PLoS ONE, 7(4)
Facioscapulohumeral Disease (FSHD) is a dominantly inherited progressive myopathy associated with aberrant production of the transcription factor, Double Homeobox Protein 4 (DUX4). The expression of DUX4 depends on an open chromatin conformation of t
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::fcefa02136a54d7c4795ebe0b3caf0de
https://hdl.handle.net/1887/98007
https://hdl.handle.net/1887/98007
Publikováno v:
Molecular therapy : the journal of the American Society of Gene Therapy. 18(9)
Inherited skin blistering conditions collectively named epidermolysis bullosa (EB) cause significant morbidity and mortality due to the compromise of the skin's barrier function, the pain of blisters, inflammation, and in some cases scaring and cance