Matches in SemOpenAlex for { <https://semopenalex.org/work/W2144559222> ?p ?o ?g. }
- W2144559222 endingPage "1374" @default.
- W2144559222 startingPage "1365" @default.
- W2144559222 abstract "Introduction: Facioscapulohumeral muscular dystrophy (FSHD) is an autosomal dominant genetic disorder characterized by progressive muscle degeneration. Currently, no effective treatment exists for the disease. Although the causative gene for FSHD, the double homeobox protein 4 (DUX4) gene, was identified over recent years, little effort has been made to develop targeted therapies. This is due to a lack of understanding of the gene and pathways involved. However, with the recent discovery that overexpression of myopathic DUX4 gene causes FSHD pathogenesis, inhibition of DUX4 and its downstream molecule has emerged as a promising therapeutic strategy against FSHD.Areas covered: In this paper we will discuss and review the latest research in the area of antisense knockdown therapy for FSHD, as well as a variety of accompanying issues, including efficacy and potential of antisense oligonucleotides.Expert opinion: Very recently, an effective antisense knockdown therapy targeting the paired-like homeodomain transcription factor 1 (PITX1) gene has demonstrated success in a newly developed mouse model. Similar to DUX4, PITX1 is specifically up-regulated in FSHD affected muscles. As such, the same knockdown principle using oligonucleotide could be applied to suppress the aberrantly expressed DUX4 and PITX1 in FSHD patients." @default.
- W2144559222 created "2016-06-24" @default.
- W2144559222 creator A5034122987 @default.
- W2144559222 creator A5080323628 @default.
- W2144559222 date "2015-09-24" @default.
- W2144559222 modified "2023-09-27" @default.
- W2144559222 title "Potential of antisense therapy for facioscapulohumeral muscular dystrophy" @default.
- W2144559222 cites W155685562 @default.
- W2144559222 cites W1771343264 @default.
- W2144559222 cites W1835869536 @default.
- W2144559222 cites W1963981436 @default.
- W2144559222 cites W1964930281 @default.
- W2144559222 cites W1965534780 @default.
- W2144559222 cites W1971643509 @default.
- W2144559222 cites W1979404984 @default.
- W2144559222 cites W1985659366 @default.
- W2144559222 cites W1993785908 @default.
- W2144559222 cites W1996577262 @default.
- W2144559222 cites W1997089606 @default.
- W2144559222 cites W1998062328 @default.
- W2144559222 cites W1998398326 @default.
- W2144559222 cites W1999638106 @default.
- W2144559222 cites W2000725203 @default.
- W2144559222 cites W2001021923 @default.
- W2144559222 cites W2003642545 @default.
- W2144559222 cites W2003777838 @default.
- W2144559222 cites W2003975348 @default.
- W2144559222 cites W2004474116 @default.
- W2144559222 cites W2004824383 @default.
- W2144559222 cites W2005286540 @default.
- W2144559222 cites W2009635392 @default.
- W2144559222 cites W2012621742 @default.
- W2144559222 cites W2012789654 @default.
- W2144559222 cites W2016109355 @default.
- W2144559222 cites W2017826157 @default.
- W2144559222 cites W2018380248 @default.
- W2144559222 cites W2023292280 @default.
- W2144559222 cites W2026431435 @default.
- W2144559222 cites W2026556066 @default.
- W2144559222 cites W2026685059 @default.
- W2144559222 cites W2027064784 @default.
- W2144559222 cites W2027782787 @default.
- W2144559222 cites W2028687559 @default.
- W2144559222 cites W2033618457 @default.
- W2144559222 cites W2035051422 @default.
- W2144559222 cites W2037761873 @default.
- W2144559222 cites W2038856813 @default.
- W2144559222 cites W2040738900 @default.
- W2144559222 cites W2041743270 @default.
- W2144559222 cites W2043454383 @default.
- W2144559222 cites W2043603340 @default.
- W2144559222 cites W2044567476 @default.
- W2144559222 cites W2046369777 @default.
- W2144559222 cites W2047689734 @default.
- W2144559222 cites W2049273201 @default.
- W2144559222 cites W2050229128 @default.
- W2144559222 cites W2052590535 @default.
- W2144559222 cites W2053148264 @default.
- W2144559222 cites W2053210596 @default.
- W2144559222 cites W2058027846 @default.
- W2144559222 cites W2059659618 @default.
- W2144559222 cites W2062442451 @default.
- W2144559222 cites W2064487427 @default.
- W2144559222 cites W2065186743 @default.
- W2144559222 cites W2071230707 @default.
- W2144559222 cites W2072847969 @default.
- W2144559222 cites W2073842755 @default.
- W2144559222 cites W2076732839 @default.
- W2144559222 cites W2079291490 @default.
- W2144559222 cites W2080267978 @default.
- W2144559222 cites W2082127114 @default.
- W2144559222 cites W2083922607 @default.
- W2144559222 cites W2087453865 @default.
- W2144559222 cites W2089351680 @default.
- W2144559222 cites W2092054524 @default.
- W2144559222 cites W2093618454 @default.
- W2144559222 cites W2095426368 @default.
- W2144559222 cites W2102719739 @default.
- W2144559222 cites W2103091788 @default.
- W2144559222 cites W2104541511 @default.
- W2144559222 cites W2106224752 @default.
- W2144559222 cites W2113993299 @default.
- W2144559222 cites W2116947711 @default.
- W2144559222 cites W2117224856 @default.
- W2144559222 cites W2118562875 @default.
- W2144559222 cites W2118947165 @default.
- W2144559222 cites W2122933784 @default.
- W2144559222 cites W2134753663 @default.
- W2144559222 cites W2141695132 @default.
- W2144559222 cites W2144172560 @default.
- W2144559222 cites W2145074423 @default.
- W2144559222 cites W2154112871 @default.
- W2144559222 cites W2158041351 @default.
- W2144559222 cites W2158923719 @default.
- W2144559222 cites W2166217372 @default.
- W2144559222 cites W2166341332 @default.
- W2144559222 cites W2167135407 @default.
- W2144559222 cites W2168504323 @default.