A novel canine model for Duchenne muscular dystrophy (DMD): single nucleotide deletion in DMD gene exon 20

Autor: Sara Mata López, James J. Hammond, Madison B. Rigsby, Cynthia J. Balog-Alvarez, Joe N. Kornegay, Peter P. Nghiem

Publikováno v: Skeletal Muscle, Vol 8, Iss 1, Pp 1-6 (2018)

Abstract Background Boys with Duchenne muscular dystrophy (DMD) have DMD gene mutations, with associated loss of the dystrophin protein and progressive muscle degeneration and weakness. Corticosteroids and palliative support are currently the best tr

Externí odkaz: https://doaj.org/article/eb783a37156645ac8356d2e5603c7c91

Immortalized Canine Dystrophic Myoblast Cell Lines for Development of Peptide-Conjugated Splice-Switching Oligonucleotides

Autor: Yoshitsugu Aoki, A Arzumanov, Kamel Mamchaoui, Tone Yuichiro, Rika Maruyama, Graham McClorey, Shin'ichi Takeda, Toshifumi Yokota, Yasumasa Hashimoto, Maria K. Tsoumpra, Michihiro Imamura, Vincent Mouly, Michael J. Gait, Matthew J.A. Wood, Reiko Terada

Publikováno v: Nucleic Acid Therapeutics
Nucleic Acid Therapeutics, 2021, 31 (2), pp.172-181. ⟨10.1089/nat.2020.0907⟩
Nucleic Acid Therapeutics, Mary Ann Liebert, Inc. publishers, 2021, 31 (2), pp.172-181. ⟨10.1089/nat.2020.0907⟩

Duchenne muscular dystrophy (DMD) is a severe muscle-wasting disease caused by frameshift or nonsense mutations in the DMD gene, resulting in the loss of dystrophin from muscle membranes. Exon skipping using splice-switching oligonucleotides (SSOs) r

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::37e6bb92dfaba5985c0590e408a2e368
https://hal.sorbonne-universite.fr/hal-03276095

Skipping Multiple Exons to Treat DMD—Promises and Challenges

Autor: Tejal Aslesh, Toshifumi Yokota, Rika Maruyama

Publikováno v: Biomedicines

Duchenne muscular dystrophy (DMD) is a lethal disorder caused by mutations in the DMD gene. Antisense-mediated exon-skipping is a promising therapeutic strategy that makes use of synthetic nucleic acids to skip frame-disrupting exon(s) and allows for

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::38f348fb955f94c7fe782fb30e4a516b

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