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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| Main Authors: | , , , , , , |
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| Format: | article |
| Language: | EN |
| Published: |
Nature Portfolio
2016
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| Subjects: | |
| Online Access: | https://doaj.org/article/b3804986740f4c778bdb533bfe47c56a |
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