相似文献
1
Myotonic dystrophy: approach to therapy.
Curr Opin Genet Dev. 2017 Jun;44:135-140. doi: 10.1016/j.gde.2017.03.007. Epub 2017 Apr 1.
2
Myotonic dystrophy: clinical and molecular parallels between myotonic dystrophy type 1 and type 2.
Curr Neurol Neurosci Rep. 2002 Sep;2(5):465-70. doi: 10.1007/s11910-002-0074-6.
3
Myotonic dystrophies: An update on clinical aspects, genetic, pathology, and molecular pathomechanisms.
Biochim Biophys Acta. 2015 Apr;1852(4):594-606. doi: 10.1016/j.bbadis.2014.05.019. Epub 2014 May 29.
4
Systemic Evaluation of Chimeric LNA/2'-O-Methyl Steric Blockers for Myotonic Dystrophy Type 1 Therapy.
Nucleic Acid Ther. 2020 Apr;30(2):80-93. doi: 10.1089/nat.2019.0811. Epub 2019 Dec 23.
5
Sense and Antisense DMPK RNA Foci Accumulate in DM1 Tissues during Development.
PLoS One. 2015 Sep 4;10(9):e0137620. doi: 10.1371/journal.pone.0137620. eCollection 2015.
6
Myotonic dystrophy: RNA pathogenesis comes into focus.
Am J Hum Genet. 2004 May;74(5):793-804. doi: 10.1086/383590. Epub 2004 Apr 2.
7
Fuchs' Endothelial Corneal Dystrophy and RNA Foci in Patients With Myotonic Dystrophy.
Invest Ophthalmol Vis Sci. 2017 Sep 1;58(11):4579-4585. doi: 10.1167/iovs.17-22350.
8
Antisense transcription of the myotonic dystrophy locus yields low-abundant RNAs with and without (CAG)n repeat.
RNA Biol. 2017 Oct 3;14(10):1374-1388. doi: 10.1080/15476286.2017.1279787. Epub 2017 Jan 19.
9
[Myotonic dystrophy - a new insight into a well-known disease].
Neurol Neurochir Pol. 2010 May-Jun;44(3):264-76. doi: 10.1016/s0028-3843(14)60041-4.
10
Therapeutic Genome Editing for Myotonic Dystrophy Type 1 Using CRISPR/Cas9.
Mol Ther. 2018 Nov 7;26(11):2617-2630. doi: 10.1016/j.ymthe.2018.09.003. Epub 2018 Sep 11.
引用本文的文献
1
Chemical Modifications in Nucleic Acid Therapeutics.
Methods Mol Biol. 2025;2965:57-126. doi: 10.1007/978-1-0716-4742-4_3.
2
A KIF1C-CNBP motor-adaptor complex for trafficking mRNAs to cell protrusions.
Cell Rep. 2025 Mar 25;44(3):115346. doi: 10.1016/j.celrep.2025.115346. Epub 2025 Feb 20.
3
A Novel Class of FKBP12 Ligands Rescues Premature Aging Phenotypes Associated with Myotonic Dystrophy Type 1.
Cells. 2024 Nov 22;13(23):1939. doi: 10.3390/cells13231939.
4
Erythromycin for myotonic dystrophy type 1: a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial.
EClinicalMedicine. 2023 Dec 26;67:102390. doi: 10.1016/j.eclinm.2023.102390. eCollection 2024 Jan.
5
Acute leg pain and weakness in pregnancy: A new diagnosis of myotonic dystrophy.
Obstet Med. 2023 Dec;16(4):253-255. doi: 10.1177/1753495X221109738. Epub 2022 Jun 30.
6
Muscleblind-like proteins use modular domains to localize RNAs by riding kinesins and docking to membranes.
Nat Commun. 2023 Jun 9;14(1):3427. doi: 10.1038/s41467-023-38923-6.
7
Specific -promoter targeting by CRISPRi reverses myotonic dystrophy type 1-associated defects in patient muscle cells.
Mol Ther Nucleic Acids. 2023 May 13;32:857-871. doi: 10.1016/j.omtn.2023.05.007. eCollection 2023 Jun 13.
8
Block or degrade? Balancing on- and off-target effects of antisense strategies against transcripts with expanded triplet repeats in DM1.
Mol Ther Nucleic Acids. 2023 Apr 20;32:622-636. doi: 10.1016/j.omtn.2023.04.010. eCollection 2023 Jun 13.
9
Therapeutic targeting of CNBP phase separation inhibits ribosome biogenesis and neuroblastoma progression via modulating SWI/SNF complex activity.
Clin Transl Med. 2023 Apr;13(4):e1235. doi: 10.1002/ctm2.1235.
10
Associations between lower extremity muscle fat fraction and motor performance in myotonic dystrophy type 2: A pilot study.
Muscle Nerve. 2023 Jun;67(6):506-514. doi: 10.1002/mus.27821. Epub 2023 Apr 3.
本文引用的文献
1
Targeting deregulated AMPK/mTORC1 pathways improves muscle function in myotonic dystrophy type I.
J Clin Invest. 2017 Feb 1;127(2):549-563. doi: 10.1172/JCI89616. Epub 2017 Jan 9.
2
Precise small-molecule recognition of a toxic CUG RNA repeat expansion.
Nat Chem Biol. 2017 Feb;13(2):188-193. doi: 10.1038/nchembio.2251. Epub 2016 Dec 12.
3
Mechanistic determinants of MBNL activity.
Nucleic Acids Res. 2016 Dec 1;44(21):10326-10342. doi: 10.1093/nar/gkw915. Epub 2016 Oct 12.
4
Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy.
Proc Natl Acad Sci U S A. 2016 Sep 27;113(39):10962-7. doi: 10.1073/pnas.1605731113. Epub 2016 Sep 12.
5
Dmpk gene deletion or antisense knockdown does not compromise cardiac or skeletal muscle function in mice.
Hum Mol Genet. 2016 Oct 1;25(19):4328-4338. doi: 10.1093/hmg/ddw266. Epub 2016 Aug 13.
6
Spt4 selectively regulates the expression of C9orf72 sense and antisense mutant transcripts.
Science. 2016 Aug 12;353(6300):708-12. doi: 10.1126/science.aaf7791.
7
A Potent Inhibitor of Protein Sequestration by Expanded Triplet (CUG) Repeats that Shows Phenotypic Improvements in a Drosophila Model of Myotonic Dystrophy.
ChemMedChem. 2016 Jul 5;11(13):1428-35. doi: 10.1002/cmdc.201600081. Epub 2016 Jun 1.
8
Oral administration of erythromycin decreases RNA toxicity in myotonic dystrophy.
Ann Clin Transl Neurol. 2015 Dec 10;3(1):42-54. doi: 10.1002/acn3.271. eCollection 2016 Jan.
9
Actinomycin D Specifically Reduces Expanded CUG Repeat RNA in Myotonic Dystrophy Models.
Cell Rep. 2015 Dec 22;13(11):2386-2394. doi: 10.1016/j.celrep.2015.11.028. Epub 2015 Dec 10.
10
The rates of the major steps in the molecular mechanism of RNase H1-dependent antisense oligonucleotide induced degradation of RNA.
Nucleic Acids Res. 2015 Oct 15;43(18):8955-63. doi: 10.1093/nar/gkv920. Epub 2015 Sep 17.
Zotero 插件