Morpholino oligonucleotide-mediated exon skipping for DMD treatment: Past insights, present challenges and future perspectives
Author:
Publisher
Springer Science and Business Media LLC
Subject
General Agricultural and Biological Sciences,General Biochemistry, Genetics and Molecular Biology,General Medicine
Link
https://link.springer.com/content/pdf/10.1007/s12038-023-00365-z.pdf
Reference82 articles.
1. Aartsma-Rus A 2022 Good news for the mdx mouse community: Improved dystrophin restoration after skipping mouse dystrophin exon 23. Mol. Ther. Nucleic Acids 30 355–356
2. Aartsma-Rus A, De Winter CL, Janson AA, et al. 2005 Functional analysis of 114 exon-internal AONs for targeted DMD exon skipping: indication for steric hindrance of SR protein binding sites. Oligonucleotides 15 284–197
3. Aartsma-Rus A, Morgan J, Lonkar P, et al. 2019 Report of a TREAT-NMD/world Duchenne organisation meeting on dystrophin quantification methodology. J. Neuromuscul. Dis. 6 147–159
4. Agarwal S 2020 High-dose AAV gene therapy deaths. Nat. Biotechnol. 38 910
5. Akpulat U, Wang H, Becker K, et al. 2018 Shorter phosphorodiamidate morpholino splice-switching oligonucleotides may increase exon-skipping efficacy in DMD. Mol. Ther. Nucleic Acids 13 534–542
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