Therapeutic activity of modified U1 core spliceosomal particles

Therapeutic activity of modified U1 core spliceosomal particles

Modification of the spliceosome is being tested as a potential therapy for exon-skipping diseases, such as spinal muscular atrophy (SMA). Here the authors show that 70K and stem loop IV structural elements of a modified U1 particle are essential for splicing enhancement and effective treatment of SM...

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Autores principales: Malgorzata Ewa Rogalska, Mojca Tajnik, Danilo Licastro, Erica Bussani, Luca Camparini, Chiara Mattioli, Franco Pagani
Formato: article
Lenguaje:EN
Publicado: Nature Portfolio 2016
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Science
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Acceso en línea:https://doaj.org/article/b3804986740f4c778bdb533bfe47c56a
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