226 related articles for article (PubMed ID: 35260651)
1. Transiently expressed CRISPR/Cas9 induces wild-type dystrophin in vitro in DMD patient myoblasts carrying duplications.
Pini V; Mariot V; Dumonceaux J; Counsell J; O'Neill HC; Farmer S; Conti F; Muntoni F
Sci Rep; 2022 Mar; 12(1):3756. PubMed ID: 35260651
[TBL] [Abstract][Full Text] [Related]
2. Restoration of Dystrophin Protein Expression by Exon Skipping Utilizing CRISPR-Cas9 in Myoblasts Derived from DMD Patient iPS Cells.
Ifuku M; Iwabuchi KA; Tanaka M; Lung MSY; Hotta A
Methods Mol Biol; 2018; 1828():191-217. PubMed ID: 30171543
[TBL] [Abstract][Full Text] [Related]
3. CRISPR/Cas9-based genome editing for the modification of multiple duplications that cause Duchenne muscular dystrophy.
Wang DN; Wang ZQ; Jin M; Lin MT; Wang N
Gene Ther; 2022 Dec; 29(12):730-737. PubMed ID: 35534612
[TBL] [Abstract][Full Text] [Related]
4. Selection-free gene repair after adenoviral vector transduction of designer nucleases: rescue of dystrophin synthesis in DMD muscle cell populations.
Maggio I; Stefanucci L; Janssen JM; Liu J; Chen X; Mouly V; Gonçalves MA
Nucleic Acids Res; 2016 Feb; 44(3):1449-70. PubMed ID: 26762977
[TBL] [Abstract][Full Text] [Related]
5. CRISPR/Cas9 editing of directly reprogrammed myogenic progenitors restores dystrophin expression in a mouse model of muscular dystrophy.
Domenig SA; Bundschuh N; Lenardič A; Ghosh A; Kim I; Qabrati X; D'Hulst G; Bar-Nur O
Stem Cell Reports; 2022 Feb; 17(2):321-336. PubMed ID: 34995499
[TBL] [Abstract][Full Text] [Related]
6. Challenges associated with homologous directed repair using CRISPR-Cas9 and TALEN to edit the DMD genetic mutation in canine Duchenne muscular dystrophy.
Mata López S; Balog-Alvarez C; Vitha S; Bettis AK; Canessa EH; Kornegay JN; Nghiem PP
PLoS One; 2020; 15(1):e0228072. PubMed ID: 31961902
[TBL] [Abstract][Full Text] [Related]
7. Gene editing of Duchenne muscular dystrophy using biomineralization-based spCas9 variant nanoparticles.
Li S; Du M; Deng J; Deng G; Li J; Song Z; Han H
Acta Biomater; 2022 Dec; 154():597-607. PubMed ID: 36243370
[TBL] [Abstract][Full Text] [Related]
8. Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy.
Bengtsson NE; Hall JK; Odom GL; Phelps MP; Andrus CR; Hawkins RD; Hauschka SD; Chamberlain JR; Chamberlain JS
Nat Commun; 2017 Feb; 8():14454. PubMed ID: 28195574
[TBL] [Abstract][Full Text] [Related]
9. Molecular correction of Duchenne muscular dystrophy by splice modulation and gene editing.
Hanson B; Wood MJA; Roberts TC
RNA Biol; 2021 Jul; 18(7):1048-1062. PubMed ID: 33472516
[TBL] [Abstract][Full Text] [Related]
10. Therapeutic Applications of CRISPR/Cas for Duchenne Muscular Dystrophy.
Wong TWY; Cohn RD
Curr Gene Ther; 2017; 17(4):301-308. PubMed ID: 29173172
[TBL] [Abstract][Full Text] [Related]
11. Full-length dystrophin restoration via targeted exon integration by AAV-CRISPR in a humanized mouse model of Duchenne muscular dystrophy.
Pickar-Oliver A; Gough V; Bohning JD; Liu S; Robinson-Hamm JN; Daniels H; Majoros WH; Devlin G; Asokan A; Gersbach CA
Mol Ther; 2021 Nov; 29(11):3243-3257. PubMed ID: 34509668
[TBL] [Abstract][Full Text] [Related]
12. Creation of a Novel Humanized Dystrophic Mouse Model of Duchenne Muscular Dystrophy and Application of a CRISPR/Cas9 Gene Editing Therapy.
Young CS; Mokhonova E; Quinonez M; Pyle AD; Spencer MJ
J Neuromuscul Dis; 2017; 4(2):139-145. PubMed ID: 28505980
[TBL] [Abstract][Full Text] [Related]
13. Somatic gene editing ameliorates skeletal and cardiac muscle failure in pig and human models of Duchenne muscular dystrophy.
Moretti A; Fonteyne L; Giesert F; Hoppmann P; Meier AB; Bozoglu T; Baehr A; Schneider CM; Sinnecker D; Klett K; Fröhlich T; Rahman FA; Haufe T; Sun S; Jurisch V; Kessler B; Hinkel R; Dirschinger R; Martens E; Jilek C; Graf A; Krebs S; Santamaria G; Kurome M; Zakhartchenko V; Campbell B; Voelse K; Wolf A; Ziegler T; Reichert S; Lee S; Flenkenthaler F; Dorn T; Jeremias I; Blum H; Dendorfer A; Schnieke A; Krause S; Walter MC; Klymiuk N; Laugwitz KL; Wolf E; Wurst W; Kupatt C
Nat Med; 2020 Feb; 26(2):207-214. PubMed ID: 31988462
[TBL] [Abstract][Full Text] [Related]
14. In vivo genome editing in mouse restores dystrophin expression in Duchenne muscular dystrophy patient muscle fibers.
Chen M; Shi H; Gou S; Wang X; Li L; Jin Q; Wu H; Zhang H; Li Y; Wang L; Li H; Lin J; Guo W; Jiang Z; Yang X; Xu A; Zhu Y; Zhang C; Lai L; Li X
Genome Med; 2021 Apr; 13(1):57. PubMed ID: 33845891
[TBL] [Abstract][Full Text] [Related]
15. CRISPR-Induced Deletion with SaCas9 Restores Dystrophin Expression in Dystrophic Models In Vitro and In Vivo.
Duchêne BL; Cherif K; Iyombe-Engembe JP; Guyon A; Rousseau J; Ouellet DL; Barbeau X; Lague P; Tremblay JP
Mol Ther; 2018 Nov; 26(11):2604-2616. PubMed ID: 30195724
[TBL] [Abstract][Full Text] [Related]
16. Production of Duchenne muscular dystrophy cellular model using CRISPR-Cas9 exon deletion strategy.
Alizadeh F; Abraghan YJ; Farrokhi S; Yousefi Y; Mirahmadi Y; Eslahi A; Mojarrad M
Mol Cell Biochem; 2024 May; 479(5):1027-1040. PubMed ID: 37289342
[TBL] [Abstract][Full Text] [Related]
17. From gRNA Identification to the Restoration of Dystrophin Expression: A Dystrophin Gene Correction Strategy for Duchenne Muscular Dystrophy Mutations Using the CRISPR-Induced Deletion Method.
Duchêne B; Iyombe-Engembe JP; Rousseau J; Tremblay JP; Ouellet DL
Methods Mol Biol; 2018; 1687():267-283. PubMed ID: 29067670
[TBL] [Abstract][Full Text] [Related]
18. Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy.
Ousterout DG; Kabadi AM; Thakore PI; Majoros WH; Reddy TE; Gersbach CA
Nat Commun; 2015 Feb; 6():6244. PubMed ID: 25692716
[TBL] [Abstract][Full Text] [Related]
19. A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells.
Young CS; Hicks MR; Ermolova NV; Nakano H; Jan M; Younesi S; Karumbayaram S; Kumagai-Cresse C; Wang D; Zack JA; Kohn DB; Nakano A; Nelson SF; Miceli MC; Spencer MJ; Pyle AD
Cell Stem Cell; 2016 Apr; 18(4):533-40. PubMed ID: 26877224
[TBL] [Abstract][Full Text] [Related]
20. Correction of dystrophin expression in cells from Duchenne muscular dystrophy patients through genomic excision of exon 51 by zinc finger nucleases.
Ousterout DG; Kabadi AM; Thakore PI; Perez-Pinera P; Brown MT; Majoros WH; Reddy TE; Gersbach CA
Mol Ther; 2015 Mar; 23(3):523-32. PubMed ID: 25492562
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]