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Page 1
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
Efficacy and muscle safety assessment of fukutin-related protein gene therapy.
Benasutti H, Maricelli JW, Seto J, Hall J, Halbert C, Wicki J, Heusgen L, Purvis N, Regnier M, Lin DC, Rodgers BD, Chamberlain JS. Benasutti H, et al. Mol Ther Methods Clin Dev. 2023 Jun 4;30:65-80. doi: 10.1016/j.omtm.2023.05.022. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37361354 Free PMC article.
PCYT2-regulated lipid biosynthesis is critical to muscle health and ageing.
Cikes D, Elsayad K, Sezgin E, Koitai E, Torma F, Orthofer M, Yarwood R, Heinz LX, Sedlyarov V, Miranda ND, Taylor A, Grapentine S, Al-Murshedi F, Abot A, Weidinger A, Kutchukian C, Sanchez C, Cronin SJF, Novatchkova M, Kavirayani A, Schuetz T, Haubner B, Haas L, Hagelkruys A, Jackowski S, Kozlov AV, Jacquemond V, Knauf C, Superti-Furga G, Rullman E, Gustafsson T, McDermot J, Lowe M, Radak Z, Chamberlain JS, Bakovic M, Banka S, Penninger JM. Cikes D, et al. Nat Metab. 2023 Mar;5(3):495-515. doi: 10.1038/s42255-023-00766-2. Epub 2023 Mar 20. Nat Metab. 2023. PMID: 36941451
Micro-dystrophin gene therapy demonstrates long-term cardiac efficacy in a severe Duchenne muscular dystrophy model.
Piepho AB, Lowe J, Cumby LR, Dorn LE, Lake DM, Rastogi N, Gertzen MD, Sturgill SL, Odom GL, Ziolo MT, Accornero F, Chamberlain JS, Rafael-Fortney JA. Piepho AB, et al. Mol Ther Methods Clin Dev. 2023 Feb 9;28:344-354. doi: 10.1016/j.omtm.2023.02.001. eCollection 2023 Mar 9. Mol Ther Methods Clin Dev. 2023. PMID: 36874243 Free PMC article.
37 results