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- W2791177166 abstract "Background and purpose: Duchenne muscular dystrophy (DMD) is a lethal progressive pediatric muscle disorder and genetically inherited as an X-linked disease that caused by mutations in the dystrophin gene. DMD leads to progressive muscle weakness, degeneration, and wasting; finally, follows with the premature demise in affected individuals due to respiratory and/or cardiac failure typically by age of 30. For decades, scientists tried massively to find an effective therapy method, but there is no absolute cure currently for patients with DMD, nevertheless, recent advanced progressions on the treatment of DMD will be hopeful in the future. Several promising gene therapies are currently under investigation. These include gene replacement, exon skipping, suppression of stop codons. More recently, a promising gene editing tool referred to as CRISPR/Cas9 offers exciting perspectives for restoring dystrophin expression in patients with DMD. This review intents to briefly describe these methods and comment on their advances. Since DMD is a genetic disorder, it should be treated by replacing the deficient DMD copy with a functional one. However, there are different types of mutations in this gene, so such therapeutic approaches are highly mutation specific and thus are personalized. Therefore, DMD has arisen as a model of genetic disorder for understanding and overcoming of the challenges of developing personalized genetic medicines, consequently, the lessons learned from these approaches will be applicable to many other disorders.Conclusions: This review provides an update on the recent gene therapies for DMD that aim to compensate for dystrophin deficiency and the related clinical trials." @default.
- W2791177166 created "2018-03-29" @default.
- W2791177166 creator A5027017805 @default.
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- W2791177166 date "2018-02-05" @default.
- W2791177166 modified "2023-10-01" @default.
- W2791177166 title "Duchenne muscular dystrophy: an updated review of common available therapies" @default.
- W2791177166 cites W1022442599 @default.
- W2791177166 cites W1498253927 @default.
- W2791177166 cites W1537646912 @default.
- W2791177166 cites W1584209487 @default.
- W2791177166 cites W1592513071 @default.
- W2791177166 cites W1595560766 @default.
- W2791177166 cites W1850477203 @default.
- W2791177166 cites W1900565974 @default.
- W2791177166 cites W1971156867 @default.
- W2791177166 cites W1974794264 @default.
- W2791177166 cites W1975544443 @default.
- W2791177166 cites W1978686886 @default.
- W2791177166 cites W1987745724 @default.
- W2791177166 cites W1996788258 @default.
- W2791177166 cites W1997327853 @default.
- W2791177166 cites W1997340271 @default.
- W2791177166 cites W1998177003 @default.
- W2791177166 cites W1998398326 @default.
- W2791177166 cites W2002153634 @default.
- W2791177166 cites W2005330089 @default.
- W2791177166 cites W2006424459 @default.
- W2791177166 cites W2009324880 @default.
- W2791177166 cites W2009858080 @default.
- W2791177166 cites W2010417109 @default.
- W2791177166 cites W2014639769 @default.
- W2791177166 cites W2018205762 @default.
- W2791177166 cites W2029903254 @default.
- W2791177166 cites W2031126348 @default.
- W2791177166 cites W2036176224 @default.
- W2791177166 cites W2037931223 @default.
- W2791177166 cites W2038461956 @default.
- W2791177166 cites W2040578546 @default.
- W2791177166 cites W2041927972 @default.
- W2791177166 cites W2042186220 @default.
- W2791177166 cites W2046549076 @default.
- W2791177166 cites W2047563414 @default.
- W2791177166 cites W2058807250 @default.
- W2791177166 cites W2062078686 @default.
- W2791177166 cites W2062442451 @default.
- W2791177166 cites W2063331435 @default.
- W2791177166 cites W2066367443 @default.
- W2791177166 cites W2066950610 @default.
- W2791177166 cites W2067671642 @default.
- W2791177166 cites W2073293518 @default.
- W2791177166 cites W2076333986 @default.
- W2791177166 cites W2077583368 @default.
- W2791177166 cites W2078616377 @default.
- W2791177166 cites W2078966715 @default.
- W2791177166 cites W2080324830 @default.
- W2791177166 cites W2083756165 @default.
- W2791177166 cites W2089484241 @default.
- W2791177166 cites W2090849645 @default.
- W2791177166 cites W2092411055 @default.
- W2791177166 cites W2093025966 @default.
- W2791177166 cites W2093377166 @default.
- W2791177166 cites W2094835866 @default.
- W2791177166 cites W2094998522 @default.
- W2791177166 cites W2095917282 @default.
- W2791177166 cites W2097136884 @default.
- W2791177166 cites W2100682779 @default.
- W2791177166 cites W2101455790 @default.
- W2791177166 cites W2104588821 @default.
- W2791177166 cites W2104660283 @default.
- W2791177166 cites W2116021928 @default.
- W2791177166 cites W2117345872 @default.
- W2791177166 cites W2121066313 @default.
- W2791177166 cites W2121402919 @default.
- W2791177166 cites W2129171086 @default.
- W2791177166 cites W2131396113 @default.
- W2791177166 cites W2140857334 @default.
- W2791177166 cites W2141695132 @default.
- W2791177166 cites W2142261920 @default.
- W2791177166 cites W2146295849 @default.
- W2791177166 cites W2148034459 @default.
- W2791177166 cites W2153507951 @default.
- W2791177166 cites W2157085976 @default.
- W2791177166 cites W2166217372 @default.
- W2791177166 cites W2175838118 @default.
- W2791177166 cites W2188223075 @default.
- W2791177166 cites W2196662084 @default.
- W2791177166 cites W2202180511 @default.
- W2791177166 cites W2203460689 @default.
- W2791177166 cites W2205893844 @default.
- W2791177166 cites W2208696160 @default.
- W2791177166 cites W2210379934 @default.