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Page 1
Facioscapulohumeral muscular dystrophy: the road to targeted therapies.
Tihaya MS, Mul K, Balog J, de Greef JC, Tapscott SJ, Tawil R, Statland JM, van der Maarel SM. Tihaya MS, et al. Nat Rev Neurol. 2023 Feb;19(2):91-108. doi: 10.1038/s41582-022-00762-2. Epub 2023 Jan 10. Nat Rev Neurol. 2023. PMID: 36627512 Free PMC article. Review.
Split intein-mediated protein trans-splicing to express large dystrophins.
Tasfaout H, Halbert CL, McMillen TS, Allen JM, Reyes TR, Flint GV, Grimm D, Hauschka SD, Regnier M, Chamberlain JS. Tasfaout H, et al. Nature. 2024 Aug;632(8023):192-200. doi: 10.1038/s41586-024-07710-8. Epub 2024 Jul 17. Nature. 2024. PMID: 39020181 Free PMC article.
Regional and bilateral MRI and gene signatures in facioscapulohumeral dystrophy: implications for clinical trial design and mechanisms of disease progression.
Wong CJ, Friedman SD, Snider L, Bennett SR, Jones TI, Jones PL, Shaw DWW, Blemker SS, Riem L, DuCharme O, Lemmers RJFL, van der Maarel SM, Wang LH, Tawil R, Statland JM, Tapscott SJ. Wong CJ, et al. Hum Mol Genet. 2024 Apr 8;33(8):698-708. doi: 10.1093/hmg/ddae007. Hum Mol Genet. 2024. PMID: 38268317
Suspended Tissue Open Microfluidic Patterning (STOMP).
Haack AJ, Brown LG, Goldstein AJ, Mulimani P, Berthier J, Viswanathan AR, Kopyeva I, Whitten JM, Lin A, Nguyen SH, Leahy TP, Bouker EE, Padgett RM, Mazzawi NA, Tokihiro JC, Bretherton RC, Wu A, Tapscott SJ, DeForest CA, Popowics TE, Berthier E, Sniadecki NJ, Theberge AB. Haack AJ, et al. bioRxiv [Preprint]. 2024 Oct 7:2024.10.04.616662. doi: 10.1101/2024.10.04.616662. bioRxiv. 2024. PMID: 39416011 Free PMC article. Preprint.
32 results