TERAPIA EXON SKIPPING

PER LE SARCOGLICANOPATIE


A gene-edited mouse model of limb-girdle muscular dystrophy 2C for testing exon skipping

| Terapia Exon Skipping

Dis Model Mech. 2019 Nov 4;13(2):dmm040832. doi: 10.1242/dmm.040832.

Alexis R Demonbreun 1 2, Eugene J Wyatt 1, Katherine S Fallon 1, Claire C Oosterbaan 1, Patrick G Page 1,
Michele Hadhazy 1, Mattia Quattrocelli 1, David Y Barefield 1, Elizabeth M McNally 3


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PMID: 31582396 PMCID: PMC6906631 DOI: 10.1242/dmm.040832
Free PMC article


https://pubmed.ncbi.nlm.nih.gov/31582396/

Recent advancements in exon-skipping therapies using antisense oligonucleotides and genome editing for the treatment of various muscular dystrophies

| Terapia Exon Skipping

Expert Rev Mol Med . 2019 Oct 2;21:e5. doi: 10.1017/erm.2019.5.

Jaeho Hwang 1, Toshifumi Yokota 1 2
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PMID: 31576784 DOI: 10.1017/erm.2019.5
https://pubmed.ncbi.nlm.nih.gov/31576784/

Efficient exon skipping of SGCG mutations mediated by phosphorodiamidate morpholino oligomers

| Terapia Exon Skipping

JCI Insight . 2018 May 3;3(9):e99357. doi: 10.1172/jci.insight.99357.

Eugene J Wyatt 1, Alexis R Demonbreun 1, Ellis Y Kim 2, Megan J Puckelwartz 1, Andy H Vo 3, Lisa M Dellefave-Castillo 1, Quan Q Gao 4, Mariz Vainzof 5, Rita C M Pavanello 5, Mayana Zatz 5, Elizabeth M McNally 1


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PMID: 29720576 PMCID: PMC6012523 DOI: 10.1172/jci.insight.99357

Free PMC article
https://pubmed.ncbi.nlm.nih.gov/29720576/

Designing Effective Antisense Oligonucleotides for Exon Skipping

| Terapia Exon Skipping

Methods Mol Biol. 2018;1687:143-155. doi: 10.1007/978-1-4939-7374-3_10.

Takenori Shimo 1 2, Rika Maruyama 1, Toshifumi Yokota 3 4


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PMID: 29067661 DOI: 10.1007/978-1-4939-7374-3_10
https://pubmed.ncbi.nlm.nih.gov/29067661/

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