Increase in Full-Length Dystrophin by Exon Skipping in Duchenne Muscular Dystrophy Patients with Single Exon Duplications: An Open-label Study

Autor: Nicolau, Stefan, Malhotra, Jyoti, Kaler, Maryann, Magistrado-Coxen, Pamela, Iammarino, Megan A., Reash, Natalie F., Frair, Emma C., Wijeratne, Saranga, Kelly, Benjamin J., White, Peter, Lowes, Linda P., Waldrop, Megan A., Flanigan, Kevin M.

Publikováno v: Journal of Neuromuscular Diseases; April 2024, Vol. 11 Issue: 3 p679-685, 7p

The most abundant cyst wall proteins of Acanthamoeba castellanii are lectins that bind cellulose and localize to distinct structures in developing and mature cyst walls.

Autor: Pamela Magistrado-Coxen, Yousuf Aqeel, Angelo Lopez, John R Haserick, Breeanna R Urbanowicz, Catherine E Costello, John Samuelson

Publikováno v: PLoS Neglected Tropical Diseases, Vol 13, Iss 5, p e0007352 (2019)

BackgroundAcanthamoeba castellanii, which causes keratitis and blindness in under-resourced countries, is an emerging pathogen worldwide, because of its association with contact lens use. The wall makes cysts resistant to sterilizing reagents in lens

Externí odkaz: https://doaj.org/article/614497fdf9cd46d5ac998e60b7f4b006

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424P Micro-dystrophin expression and safety with delandistrogene moxeparvovec gene therapy for DMD in a broad population: phase 1B trial (ENDEAVOR).

Autor: Proud, C.¹ (AUTHOR), Zaidman, C.² (AUTHOR), McDonald, C.³ (AUTHOR), Day, J.⁴ (AUTHOR), Thrasher, P.⁵ (AUTHOR), Asher, D.⁵ (AUTHOR), Murphy, A.⁶ (AUTHOR), Guridi, M.⁷ (AUTHOR), Ding, K.⁵ (AUTHOR), Reid, C.⁶ (AUTHOR), Lewis, S.⁵ (AUTHOR), Magistrado-Coxen, P.⁵ (AUTHOR), Palatinsky, E.⁵ (AUTHOR), Wandel, C.⁷ (AUTHOR), Potter, R.⁵ (AUTHOR), Rodino-Klapac, L.⁵ (AUTHOR), Mendell, J.^8,9 (AUTHOR)

Publikováno v: Neuromuscular Disorders. 2024 Supplement 1, Vol. 43, pN.PAG-N.PAG. 1p.