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Journal Abstract Search
224 related items for PubMed ID: 34131248
1. CRISPR mediated targeting of DUX4 distal regulatory element represses DUX4 target genes dysregulated in Facioscapulohumeral muscular dystrophy. Das S, Chadwick BP. Sci Rep; 2021 Jun 15; 11(1):12598. PubMed ID: 34131248 [Abstract] [Full Text] [Related]
2. CRISPR/dCas9-mediated Transcriptional Inhibition Ameliorates the Epigenetic Dysregulation at D4Z4 and Represses DUX4-fl in FSH Muscular Dystrophy. Himeda CL, Jones TI, Jones PL. Mol Ther; 2016 Mar 15; 24(3):527-35. PubMed ID: 26527377 [Abstract] [Full Text] [Related]
3. Facioscapulohumeral muscular dystrophy and DUX4: breaking the silence. van der Maarel SM, Tawil R, Tapscott SJ. Trends Mol Med; 2011 May 15; 17(5):252-8. PubMed ID: 21288772 [Abstract] [Full Text] [Related]
8. Facioscapulohumeral muscular dystrophy: molecular pathological advances and future directions. Statland JM, Tawil R. Curr Opin Neurol; 2011 Oct 15; 24(5):423-8. PubMed ID: 21734574 [Abstract] [Full Text] [Related]
9. Intronic SMCHD1 variants in FSHD: testing the potential for CRISPR-Cas9 genome editing. Goossens R, van den Boogaard ML, Lemmers RJLF, Balog J, van der Vliet PJ, Willemsen IM, Schouten J, Maggio I, van der Stoep N, Hoeben RC, Tapscott SJ, Geijsen N, Gonçalves MAFV, Sacconi S, Tawil R, van der Maarel SM. J Med Genet; 2019 Dec 15; 56(12):828-837. PubMed ID: 31676591 [Abstract] [Full Text] [Related]
11. Applying genome-wide CRISPR-Cas9 screens for therapeutic discovery in facioscapulohumeral muscular dystrophy. Lek A, Zhang Y, Woodman KG, Huang S, DeSimone AM, Cohen J, Ho V, Conner J, Mead L, Kodani A, Pakula A, Sanjana N, King OD, Jones PL, Wagner KR, Lek M, Kunkel LM. Sci Transl Med; 2020 Mar 25; 12(536):. PubMed ID: 32213627 [Abstract] [Full Text] [Related]
12. A patient-derived iPSC model revealed oxidative stress increases facioscapulohumeral muscular dystrophy-causative DUX4. Sasaki-Honda M, Jonouchi T, Arai M, Hotta A, Mitsuhashi S, Nishino I, Matsuda R, Sakurai H. Hum Mol Genet; 2018 Dec 01; 27(23):4024-4035. PubMed ID: 30107443 [Abstract] [Full Text] [Related]