相似文献
1
Cardiac Myoediting Attenuates Cardiac Abnormalities in Human and Mouse Models of Duchenne Muscular Dystrophy.
Circ Res. 2021 Sep 3;129(6):602-616. doi: 10.1161/CIRCRESAHA.121.319579. Epub 2021 Aug 10.
2
In Vivo Genome Editing Restores Dystrophin Expression and Cardiac Function in Dystrophic Mice.
Circ Res. 2017 Sep 29;121(8):923-929. doi: 10.1161/CIRCRESAHA.117.310996. Epub 2017 Aug 8.
3
Correction of Three Prominent Mutations in Mouse and Human Models of Duchenne Muscular Dystrophy by Single-Cut Genome Editing.
Mol Ther. 2020 Sep 2;28(9):2044-2055. doi: 10.1016/j.ymthe.2020.05.024. Epub 2020 May 30.
4
Life-Long AAV-Mediated CRISPR Genome Editing in Dystrophic Heart Improves Cardiomyopathy without Causing Serious Lesions in mdx Mice.
Mol Ther. 2019 Aug 7;27(8):1407-1414. doi: 10.1016/j.ymthe.2019.05.001. Epub 2019 May 15.
5
CRISPR-Cas9 corrects Duchenne muscular dystrophy exon 44 deletion mutations in mice and human cells.
Sci Adv. 2019 Mar 6;5(3):eaav4324. doi: 10.1126/sciadv.aav4324. eCollection 2019 Mar.
6
Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy.
Nat Med. 2019 Mar;25(3):427-432. doi: 10.1038/s41591-019-0344-3. Epub 2019 Feb 18.
7
Targeted addition of mini-dystrophin into rDNA locus of Duchenne muscular dystrophy patient-derived iPSCs.
Biochem Biophys Res Commun. 2021 Mar 19;545:40-45. doi: 10.1016/j.bbrc.2021.01.056. Epub 2021 Feb 1.
8
Therapeutic Applications of CRISPR/Cas for Duchenne Muscular Dystrophy.
Curr Gene Ther. 2017;17(4):301-308. doi: 10.2174/1566523217666171121165046.
9
Micro-dystrophin gene therapy prevents heart failure in an improved Duchenne muscular dystrophy cardiomyopathy mouse model.
JCI Insight. 2021 Apr 8;6(7):146511. doi: 10.1172/jci.insight.146511.
10
Systemic administration of micro-dystrophin restores cardiac geometry and prevents dobutamine-induced cardiac pump failure.
Mol Ther. 2007 Jun;15(6):1086-92. doi: 10.1038/sj.mt.6300144. Epub 2007 Apr 17.
引用本文的文献
1
Genomic Editing of a Pathogenic Mutation in ACTA2 Rescues Multisystemic Smooth Muscle Dysfunction Syndrome in Mice.
Circulation. 2025 May 16. doi: 10.1161/CIRCULATIONAHA.125.074218.
2
The Potential of RNA Therapeutics in Treating Cardiovascular Disease.
Drugs. 2025 May;85(5):659-676. doi: 10.1007/s40265-025-02173-1. Epub 2025 Apr 2.
3
Current Advances and Future Directions of Pluripotent Stem Cells-Derived Engineered Heart Tissue for Treatment of Cardiovascular Diseases.
Cells. 2024 Dec 18;13(24):2098. doi: 10.3390/cells13242098.
4
Ryanodine receptor dysfunction causes senescence and fibrosis in Duchenne dilated cardiomyopathy.
J Cachexia Sarcopenia Muscle. 2024 Apr;15(2):536-551. doi: 10.1002/jcsm.13411. Epub 2024 Jan 14.
5
Efficacy of exon-skipping therapy for DMD cardiomyopathy with mutations in actin binding domain 1.
Mol Ther Nucleic Acids. 2023 Oct 19;34:102060. doi: 10.1016/j.omtn.2023.102060. eCollection 2023 Dec 12.
6
Recent advances on the role of monoamine oxidases in cardiac pathophysiology.
Basic Res Cardiol. 2023 Oct 4;118(1):41. doi: 10.1007/s00395-023-01012-2.
7
Elimination of CaMKIIδ Autophosphorylation by CRISPR-Cas9 Base Editing Improves Survival and Cardiac Function in Heart Failure in Mice.
Circulation. 2023 Nov 7;148(19):1490-1504. doi: 10.1161/CIRCULATIONAHA.123.065117. Epub 2023 Sep 15.
8
Modeling Duchenne Muscular Dystrophy Cardiomyopathy with Patients' Induced Pluripotent Stem-Cell-Derived Cardiomyocytes.
Int J Mol Sci. 2023 May 12;24(10):8657. doi: 10.3390/ijms24108657.
9
Single-swap editing for the correction of common Duchenne muscular dystrophy mutations.
Mol Ther Nucleic Acids. 2023 Apr 19;32:522-535. doi: 10.1016/j.omtn.2023.04.009. eCollection 2023 Jun 13.
10
Base editing correction of hypertrophic cardiomyopathy in human cardiomyocytes and humanized mice.
Nat Med. 2023 Feb;29(2):401-411. doi: 10.1038/s41591-022-02176-5. Epub 2023 Feb 16.
本文引用的文献
1
The Complex and Diverse Genetic Architecture of Dilated Cardiomyopathy.
Circ Res. 2021 May 14;128(10):1514-1532. doi: 10.1161/CIRCRESAHA.121.318157. Epub 2021 May 13.
2
Unfolded Protein Response as a Compensatory Mechanism and Potential Therapeutic Target in PLN R14del Cardiomyopathy.
Circulation. 2021 Aug 3;144(5):382-392. doi: 10.1161/CIRCULATIONAHA.120.049844. Epub 2021 Apr 30.
3
Tubulin acetylation increases cytoskeletal stiffness to regulate mechanotransduction in striated muscle.
J Gen Physiol. 2021 Jul 5;153(7). doi: 10.1085/jgp.202012743.
4
Micro-dystrophin gene therapy prevents heart failure in an improved Duchenne muscular dystrophy cardiomyopathy mouse model.
JCI Insight. 2021 Apr 8;6(7):146511. doi: 10.1172/jci.insight.146511.
5
Essential roles of the dystrophin-glycoprotein complex in different cardiac pathologies.
Adv Med Sci. 2021 Mar;66(1):52-71. doi: 10.1016/j.advms.2020.12.004. Epub 2020 Dec 30.
6
Cell-Type-Specific Gene Regulatory Networks Underlying Murine Neonatal Heart Regeneration at Single-Cell Resolution.
Cell Rep. 2020 Dec 8;33(10):108472. doi: 10.1016/j.celrep.2020.108472.
7
Degenerative and regenerative pathways underlying Duchenne muscular dystrophy revealed by single-nucleus RNA sequencing.
Proc Natl Acad Sci U S A. 2020 Nov 24;117(47):29691-29701. doi: 10.1073/pnas.2018391117. Epub 2020 Nov 4.
8
Protocol for Single-Nucleus Transcriptomics of Diploid and Tetraploid Cardiomyocytes in Murine Hearts.
STAR Protoc. 2020 Jun 8;1(2):100049. doi: 10.1016/j.xpro.2020.100049. eCollection 2020 Sep 18.
9
Development of a Cardiac Sarcomere Functional Genomics Platform to Enable Scalable Interrogation of Human Variants.
Circulation. 2020 Dec 8;142(23):2262-2275. doi: 10.1161/CIRCULATIONAHA.120.047999. Epub 2020 Oct 7.
10
A novel mouse model of Duchenne muscular dystrophy carrying a multi-exonic deletion exhibits progressive muscular dystrophy and early-onset cardiomyopathy.
Dis Model Mech. 2020 Sep 21;13(9):dmm045369. doi: 10.1242/dmm.045369.
Zotero 插件