Antisense strategies targeting DUX4 and DUX4c as therapeutic approaches for facioscapulohumeral muscular dystrophy (FSHD)

Vanderplanck C.; Tassin A.; Ansseau E.; Lancelot C.; Derenne A.; Conotte S.; Dudome V.; Leroy B.; Wilton S.; Laoudj-Chenivesse D.; Wattiez R.; Legrand A.; Belayew A.; Coppee F.

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Antisense strategies targeting DUX4 and DUX4c as therapeutic approaches for facioscapulohumeral muscular dystrophy (FSHD)

机译：针对DUX4和DUX4c的反义策略，作为治疗面肩肱型肌营养不良症（FSHD）的方法

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《Neuromuscular disorders: NMD》 |2015年第2期|共2页
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Vanderplanck C.; Tassin A.; Ansseau E.; Lancelot C.; Derenne A.; Conotte S.; Dudome V.; Leroy B.; Wilton S.; Laoudj-Chenivesse D.; Wattiez R.; Legrand A.; Belayew A.; Coppee F.;
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正文语种 eng
中图分类神经病学;
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1. Antisense strategies targeting DUX4 and DUX4c as therapeutic approaches for facioscapulohumeral muscular dystrophy (FSHD) [J] . Vanderplanck C., Tassin A., Ansseau E., Neuromuscular disorders: NMD . 2015,第Suppla2期

机译：针对DUX4和DUX4c的反义策略，作为治疗面肩肱型肌营养不良症（FSHD）的方法
2. Antisense targeting of 3 ' end elements involved in DUX4 mRNA processing is an efficient therapeutic strategy for facioscapulohumeral dystrophy: a new gene-silencing approach [J] . Marsollier Anne-Charlotte, Ciszewski Lukasz, Mariot Virginie, Human Molecular Genetics . 2016,第8期

机译：反义靶向DUX4 mRNA加工中涉及的3'末端元件是一种有效的治疗面肩肱型营养不良的策略：一种新的基因沉默方法
3. CRISPR screen identifies therapeutic target for Facioscapulohumeral Muscular Dystrophy (FSHD) [J] . Genetics in medicine . 2020,第6期

机译：CRISPR筛网识别面部鞘膜环肌营养不良症（FSHD）的治疗靶标
4. Challenges for antisense oligonucleotide-based therapeutics, in particular for exon 51-skipping in Duchenne muscular dystrophy [C] . Aoki Yoshitsugu, Nagata Tetsuya, Shimizu Yuko, 2011 Fourth International Conference on Modeling, Simulation and Applied Optimization . 2011

机译：反义寡核苷酸疗法的挑战，尤其是杜氏肌营养不良症中外显子51跳跃的挑战
5. Gene Therapy for Facioscapulohumeral Muscular Dystrophy. [D] . Wallace, Lindsay M. 2012

机译：面肩肱型肌营养不良症的基因治疗。
6. Antisense Oligonucleotides Used to Target the DUX4 mRNA as Therapeutic Approaches in FaciosScapuloHumeral Muscular Dystrophy (FSHD) [O] . Eugénie Ansseau, Céline Vanderplanck, Armelle Wauters, 2017

机译：用于靶向DUX4 mRNA的反义寡核苷酸作为面部肩cap肱肱型肌营养不良症（FSHD）的治疗方法
7. Antisense Oligonucleotides Used to Target the DUX4 mRNA as Therapeutic Approaches in FaciosScapuloHumeral Muscular Dystrophy (FSHD) [O] . Eugénie Ansseau, Céline Vanderplanck, Armelle Wauters, 2017

机译：反义寡核苷酸用于靶向DUX4 mRNa作为Faciosscapulo数字肌肉营养不良（FsHD）的治疗方法

Antisense strategies targeting DUX4 and DUX4c as therapeutic approaches for facioscapulohumeral muscular dystrophy (FSHD)

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