Combining Engineered U1 snRNA and Antisense Oligonucleotides to Improve the Treatment of a BBS1 Splice Site Mutation
Manipulation of pre-mRNA processing is a promising approach toward overcoming disease-causing mutations and treating human diseases. We show that a combined treatment applying two splice-manipulating technologies improves therapeutic efficacies to correct mutation-induced splice defects. Previously,...
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| Main Authors: | , , , , |
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| Format: | Book |
| Published: |
Elsevier,
2019-12-01T00:00:00Z.
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| Subjects: | |
| Online Access: | Connect to this object online. |
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