403 related articles for article (PubMed ID: 27845387)
21. Enhanced CRISPR-Cas9 correction of Duchenne muscular dystrophy in mice by a self-complementary AAV delivery system.
Zhang Y; Li H; Min YL; Sanchez-Ortiz E; Huang J; Mireault AA; Shelton JM; Kim J; Mammen PPA; Bassel-Duby R; Olson EN
Sci Adv; 2020 Feb; 6(8):eaay6812. PubMed ID: 32128412
[TBL] [Abstract][Full Text] [Related]
22. 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]
23. Restoration of dystrophin expression and correction of Duchenne muscular dystrophy by genome editing.
Aslesh T; Erkut E; Yokota T
Expert Opin Biol Ther; 2021 Aug; 21(8):1049-1061. PubMed ID: 33401973
[No Abstract] [Full Text] [Related]
24. Prevention of muscular dystrophy in mice by CRISPR/Cas9-mediated editing of germline DNA.
Long C; McAnally JR; Shelton JM; Mireault AA; Bassel-Duby R; Olson EN
Science; 2014 Sep; 345(6201):1184-1188. PubMed ID: 25123483
[TBL] [Abstract][Full Text] [Related]
25. A novel rabbit model of Duchenne muscular dystrophy generated by CRISPR/Cas9.
Sui T; Lau YS; Liu D; Liu T; Xu L; Gao Y; Lai L; Li Z; Han R
Dis Model Mech; 2018 Jun; 11(6):. PubMed ID: 29871865
[TBL] [Abstract][Full Text] [Related]
26. 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]
27. CRISPR-Editing Therapy for Duchenne Muscular Dystrophy.
Chemello F; Olson EN; Bassel-Duby R
Hum Gene Ther; 2023 May; 34(9-10):379-387. PubMed ID: 37060194
[TBL] [Abstract][Full Text] [Related]
28. 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]
29. CRISPR Therapeutics for Duchenne Muscular Dystrophy.
Erkut E; Yokota T
Int J Mol Sci; 2022 Feb; 23(3):. PubMed ID: 35163754
[TBL] [Abstract][Full Text] [Related]
30. CRISPR-Cas9 corrects Duchenne muscular dystrophy exon 44 deletion mutations in mice and human cells.
Min YL; Li H; Rodriguez-Caycedo C; Mireault AA; Huang J; Shelton JM; McAnally JR; Amoasii L; Mammen PPA; Bassel-Duby R; Olson EN
Sci Adv; 2019 Mar; 5(3):eaav4324. PubMed ID: 30854433
[TBL] [Abstract][Full Text] [Related]
31. Role of CRISPR/Cas9 in the treatment of Duchenne muscular dystrophy and its delivery strategies.
Agrawal P; Harish V; Mohd S; Singh SK; Tewari D; Tatiparthi R; Harshita ; Vishwas S; Sutrapu S; Dua K; Gulati M
Life Sci; 2023 Oct; 330():122003. PubMed ID: 37544379
[TBL] [Abstract][Full Text] [Related]
32. 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]
33. CRISPR/Cas9 delivery with one single adenoviral vector devoid of all viral genes.
Ehrke-Schulz E; Schiwon M; Leitner T; Dávid S; Bergmann T; Liu J; Ehrhardt A
Sci Rep; 2017 Dec; 7(1):17113. PubMed ID: 29215041
[TBL] [Abstract][Full Text] [Related]
34. CRISPR technologies for the treatment of Duchenne muscular dystrophy.
Choi E; Koo T
Mol Ther; 2021 Nov; 29(11):3179-3191. PubMed ID: 33823301
[TBL] [Abstract][Full Text] [Related]
35. Dystrophin Gene-Editing Stability Is Dependent on Dystrophin Levels in Skeletal but Not Cardiac Muscles.
Bengtsson NE; Tasfaout H; Hauschka SD; Chamberlain JS
Mol Ther; 2021 Mar; 29(3):1070-1085. PubMed ID: 33160075
[TBL] [Abstract][Full Text] [Related]
36. Integrating gene delivery and gene-editing technologies by adenoviral vector transfer of optimized CRISPR-Cas9 components.
Maggio I; Zittersteijn HA; Wang Q; Liu J; Janssen JM; Ojeda IT; van der Maarel SM; Lankester AC; Hoeben RC; Gonçalves MAFV
Gene Ther; 2020 May; 27(5):209-225. PubMed ID: 31900423
[TBL] [Abstract][Full Text] [Related]
37. The gRNA Vector Level Determines the Outcome of Systemic AAV CRISPR Therapy for Duchenne Muscular Dystrophy.
Wasala NB; Million ED; Watkins TB; Wasala LP; Han J; Yue Y; Lu B; Chen SJ; Hakim CH; Duan D
Hum Gene Ther; 2022 May; 33(9-10):518-528. PubMed ID: 35350865
[TBL] [Abstract][Full Text] [Related]
38. New advancements in CRISPR based gene therapy of Duchenne muscular dystrophy.
Eslahi A; Alizadeh F; Avan A; Ferns GA; Moghbeli M; Reza Abbaszadegan M; Mojarrad M
Gene; 2023 May; 867():147358. PubMed ID: 36914142
[TBL] [Abstract][Full Text] [Related]
39. A Novel CRISPR-Cas9 Strategy to Target DYSTROPHIN Mutations Downstream of Exon 44 in Patient-Specific DMD iPSCs.
Dhoke NR; Kim H; Azzag K; Crist SB; Kiley J; Perlingeiro RCR
Cells; 2024 Jun; 13(11):. PubMed ID: 38891104
[TBL] [Abstract][Full Text] [Related]
40. In vivo non-invasive monitoring of dystrophin correction in a new Duchenne muscular dystrophy reporter mouse.
Amoasii L; Li H; Zhang Y; Min YL; Sanchez-Ortiz E; Shelton JM; Long C; Mireault AA; Bhattacharyya S; McAnally JR; Bassel-Duby R; Olson EN
Nat Commun; 2019 Oct; 10(1):4537. PubMed ID: 31586095
[TBL] [Abstract][Full Text] [Related]
[Previous] [Next] [New Search]