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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Acceso en línea:https://doaj.org/article/b3804986740f4c778bdb533bfe47c56a
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spelling oai:doaj.org-article:b3804986740f4c778bdb533bfe47c56a2021-12-02T15:33:48ZTherapeutic activity of modified U1 core spliceosomal particles10.1038/ncomms111682041-1723https://doaj.org/article/b3804986740f4c778bdb533bfe47c56a2016-04-01T00:00:00Zhttps://doi.org/10.1038/ncomms11168https://doaj.org/toc/2041-1723Modification 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 SMA mice.Malgorzata Ewa RogalskaMojca TajnikDanilo LicastroErica BussaniLuca CampariniChiara MattioliFranco PaganiNature PortfolioarticleScienceQENNature Communications, Vol 7, Iss 1, Pp 1-13 (2016)
institution DOAJ
collection DOAJ
language EN
topic Science
Q
spellingShingle Science
Q
Malgorzata Ewa Rogalska
Mojca Tajnik
Danilo Licastro
Erica Bussani
Luca Camparini
Chiara Mattioli
Franco Pagani
Therapeutic activity of modified U1 core spliceosomal particles
description 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 SMA mice.
format article
author Malgorzata Ewa Rogalska
Mojca Tajnik
Danilo Licastro
Erica Bussani
Luca Camparini
Chiara Mattioli
Franco Pagani
author_facet Malgorzata Ewa Rogalska
Mojca Tajnik
Danilo Licastro
Erica Bussani
Luca Camparini
Chiara Mattioli
Franco Pagani
author_sort Malgorzata Ewa Rogalska
title Therapeutic activity of modified U1 core spliceosomal particles
title_short Therapeutic activity of modified U1 core spliceosomal particles
title_full Therapeutic activity of modified U1 core spliceosomal particles
title_fullStr Therapeutic activity of modified U1 core spliceosomal particles
title_full_unstemmed Therapeutic activity of modified U1 core spliceosomal particles
title_sort therapeutic activity of modified u1 core spliceosomal particles
publisher Nature Portfolio
publishDate 2016
url https://doaj.org/article/b3804986740f4c778bdb533bfe47c56a
work_keys_str_mv AT malgorzataewarogalska therapeuticactivityofmodifiedu1corespliceosomalparticles
AT mojcatajnik therapeuticactivityofmodifiedu1corespliceosomalparticles
AT danilolicastro therapeuticactivityofmodifiedu1corespliceosomalparticles
AT ericabussani therapeuticactivityofmodifiedu1corespliceosomalparticles
AT lucacamparini therapeuticactivityofmodifiedu1corespliceosomalparticles
AT chiaramattioli therapeuticactivityofmodifiedu1corespliceosomalparticles
AT francopagani therapeuticactivityofmodifiedu1corespliceosomalparticles
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