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For: Li HL, Fujimoto N, Sasakawa N, Shirai S, Ohkame T, Sakuma T, Tanaka M, Amano N, Watanabe A, Sakurai H, Yamamoto T, Yamanaka S, Hotta A. Precise correction of the dystrophin gene in duchenne muscular dystrophy patient induced pluripotent stem cells by TALEN and CRISPR-Cas9. Stem Cell Reports 2014;4:143-154. [PMID: 25434822 PMCID: PMC4297888 DOI: 10.1016/j.stemcr.2014.10.013] [Citation(s) in RCA: 384] [Impact Index Per Article: 34.9] [Reference Citation Analysis] [What about the content of this article? (0)] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 09/04/2014] [Revised: 10/24/2014] [Accepted: 10/24/2014] [Indexed: 12/28/2022]  Open
Number Cited by Other Article(s)
1
Bengtsson NE, Tasfaout H, Chamberlain JS. The road toward AAV-mediated gene therapy of Duchenne muscular dystrophy. Mol Ther 2025;33:2035-2051. [PMID: 40181545 DOI: 10.1016/j.ymthe.2025.03.065] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 03/10/2025] [Revised: 03/31/2025] [Accepted: 03/31/2025] [Indexed: 04/05/2025]  Open
2
Chang J, Yang X, Zhang T, Sun H, Cheng H, Jia Z, Li Y, Ma S, Sun T, Cao J. High-Throughput Screening to Identify Novel Compounds Affecting the Genome Editing Efficiency of CRISPR System. Molecules 2025;30:1811. [PMID: 40333840 PMCID: PMC12029788 DOI: 10.3390/molecules30081811] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 02/26/2025] [Revised: 04/08/2025] [Accepted: 04/12/2025] [Indexed: 05/09/2025]  Open
3
Haque US, Yokota T. Gene Editing for Duchenne Muscular Dystrophy: From Experimental Models to Emerging Therapies. Degener Neurol Neuromuscul Dis 2025;15:17-40. [PMID: 40241992 PMCID: PMC12002074 DOI: 10.2147/dnnd.s495536] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 01/23/2025] [Accepted: 04/08/2025] [Indexed: 04/18/2025]  Open
4
He Q, Wang Y, Tan Z, Zhang X, Yu C, Jiang X. Mapping the therapeutic landscape of CRISPR-Cas9 for combating age-related diseases. Front Genome Ed 2025;7:1558432. [PMID: 40255230 PMCID: PMC12006052 DOI: 10.3389/fgeed.2025.1558432] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 01/10/2025] [Accepted: 03/19/2025] [Indexed: 04/22/2025]  Open
5
Wang AYL, Aviña AE, Liu YY, Kao HK. Pluripotent Stem Cells: Recent Advances and Emerging Trends. Biomedicines 2025;13:765. [PMID: 40299329 PMCID: PMC12025069 DOI: 10.3390/biomedicines13040765] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Track Full Text] [Download PDF] [Journal Information] [Subscribe] [Scholar Register] [Received: 03/14/2025] [Accepted: 03/19/2025] [Indexed: 04/30/2025]  Open
6
Padmaswari MH, Agrawal S, Nelson CE. Preclinical development of genome editing to treat Duchenne muscular dystrophy by exon skipping. J Neuromuscul Dis 2025:22143602251326993. [PMID: 40105473 DOI: 10.1177/22143602251326993] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 03/20/2025]
7
Kakizaki M, Hashimoto R, Nagata N, Yamamoto T, Okura T, Katoh H, Kitai Y, Akahori Y, Shirato K, Ryo A, Takayama K, Takeda M. The respective roles of TMPRSS2 and cathepsins for SARS-CoV-2 infection in human respiratory organoids. J Virol 2025;99:e0185324. [PMID: 39601592 PMCID: PMC11784140 DOI: 10.1128/jvi.01853-24] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 10/19/2024] [Accepted: 10/31/2024] [Indexed: 11/29/2024]  Open
8
Mondéjar-Parreño G, Moreno-Manuel AI, Ruiz-Robles JM, Jalife J. Ion channel traffic jams: the significance of trafficking deficiency in long QT syndrome. Cell Discov 2025;11:3. [PMID: 39788950 PMCID: PMC11717978 DOI: 10.1038/s41421-024-00738-0] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 05/07/2024] [Accepted: 09/10/2024] [Indexed: 01/12/2025]  Open
9
Mondéjar-Parreño G, Sánchez-Pérez P, Cruz FM, Jalife J. Promising tools for future drug discovery and development in antiarrhythmic therapy. Pharmacol Rev 2025;77:100013. [PMID: 39952687 DOI: 10.1124/pharmrev.124.001297] [Citation(s) in RCA: 1] [Impact Index Per Article: 1.0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 04/23/2024] [Revised: 08/30/2024] [Accepted: 10/04/2024] [Indexed: 01/22/2025]  Open
10
Azeez SS, Hamad RS, Hamad BK, Shekha MS, Bergsten P. Advances in CRISPR-Cas technology and its applications: revolutionising precision medicine. Front Genome Ed 2024;6:1509924. [PMID: 39726634 PMCID: PMC11669675 DOI: 10.3389/fgeed.2024.1509924] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 10/11/2024] [Accepted: 11/28/2024] [Indexed: 12/28/2024]  Open
11
Uno N, Miyamoto H, Yamazaki K, Egawa M, Kobayashi H, Kazuki K, Osaki M, Suzuki T, Hamamichi S, Oshimura M, Tomizuka K, Kazuki Y. Microcell-mediated chromosome transfer between non-identical human iPSCs. MOLECULAR THERAPY. NUCLEIC ACIDS 2024;35:102382. [PMID: 39634136 PMCID: PMC11616053 DOI: 10.1016/j.omtn.2024.102382] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Figures] [Subscribe] [Scholar Register] [Received: 11/26/2023] [Accepted: 10/31/2024] [Indexed: 12/07/2024]
12
Aslan C, Zolbanin NM, Faraji F, Jafari R. Exosomes for CRISPR-Cas9 Delivery: The Cutting Edge in Genome Editing. Mol Biotechnol 2024;66:3092-3116. [PMID: 38012525 DOI: 10.1007/s12033-023-00932-7] [Citation(s) in RCA: 2] [Impact Index Per Article: 2.0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 04/03/2023] [Accepted: 10/02/2023] [Indexed: 11/29/2023]
13
Lauerer AM, Caravia XM, Maier LS, Chemello F, Lebek S. Gene editing in common cardiovascular diseases. Pharmacol Ther 2024;263:108720. [PMID: 39284367 DOI: 10.1016/j.pharmthera.2024.108720] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 05/20/2024] [Revised: 07/29/2024] [Accepted: 09/01/2024] [Indexed: 09/22/2024]
14
Chaudhary R, Rehman M, Agarwal V, Kumar A, Kaushik AS, Srivastava S, Srivastava S, Verma R, Rajinikanth PS, Mishra V. Terra incognita of glial cell dynamics in the etiology of leukodystrophies: Broadening disease and therapeutic perspectives. Life Sci 2024;354:122953. [PMID: 39122110 DOI: 10.1016/j.lfs.2024.122953] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 02/05/2024] [Revised: 07/09/2024] [Accepted: 08/05/2024] [Indexed: 08/12/2024]
15
Zhao M, Taniguchi Y, Shimono C, Jonouchi T, Cheng Y, Shimizu Y, Nalbandian M, Yamamoto T, Nakagawa M, Sekiguchi K, Sakurai H. Heparan Sulfate Chain-Conjugated Laminin-E8 Fragments Advance Paraxial Mesodermal Differentiation Followed by High Myogenic Induction from hiPSCs. ADVANCED SCIENCE (WEINHEIM, BADEN-WURTTEMBERG, GERMANY) 2024;11:e2308306. [PMID: 38685581 PMCID: PMC11234437 DOI: 10.1002/advs.202308306] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Subscribe] [Scholar Register] [Received: 11/01/2023] [Revised: 03/26/2024] [Indexed: 05/02/2024]
16
Dhoke NR, Kim H, Azzag K, Crist SB, Kiley J, Perlingeiro RCR. A Novel CRISPR-Cas9 Strategy to Target DYSTROPHIN Mutations Downstream of Exon 44 in Patient-Specific DMD iPSCs. Cells 2024;13:972. [PMID: 38891104 PMCID: PMC11171783 DOI: 10.3390/cells13110972] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 05/15/2024] [Revised: 05/25/2024] [Accepted: 05/30/2024] [Indexed: 06/21/2024]  Open
17
Laurent M, Geoffroy M, Pavani G, Guiraud S. CRISPR-Based Gene Therapies: From Preclinical to Clinical Treatments. Cells 2024;13:800. [PMID: 38786024 PMCID: PMC11119143 DOI: 10.3390/cells13100800] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 03/26/2024] [Revised: 05/03/2024] [Accepted: 05/05/2024] [Indexed: 05/25/2024]  Open
18
Alizadeh F, Abraghan YJ, Farrokhi S, Yousefi Y, Mirahmadi Y, Eslahi A, Mojarrad M. Production of Duchenne muscular dystrophy cellular model using CRISPR-Cas9 exon deletion strategy. Mol Cell Biochem 2024;479:1027-1040. [PMID: 37289342 DOI: 10.1007/s11010-023-04759-3] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 03/24/2023] [Accepted: 05/03/2023] [Indexed: 06/09/2023]
19
Wang Y, Zhai Y, Zhang M, Song C, Zhang Y, Zhang G. Escaping from CRISPR-Cas-mediated knockout: the facts, mechanisms, and applications. Cell Mol Biol Lett 2024;29:48. [PMID: 38589794 PMCID: PMC11003099 DOI: 10.1186/s11658-024-00565-x] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 10/01/2023] [Accepted: 03/21/2024] [Indexed: 04/10/2024]  Open
20
Lotfi M, Ashouri A, Mojarrad M, Mozaffari-Jovin S, Abbaszadegan MR. Design Principles of a Novel Construct for HBB Gene-Editing and Investigation of Its Gene-Targeting Efficiency in HEK293 Cells. Mol Biotechnol 2024;66:517-530. [PMID: 37266832 DOI: 10.1007/s12033-023-00739-6] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 01/16/2023] [Accepted: 03/27/2023] [Indexed: 06/03/2023]
21
Saad FA, Saad JF, Siciliano G, Merlini L, Angelini C. Duchenne Muscular Dystrophy Gene Therapy. Curr Gene Ther 2024;24:17-28. [PMID: 36411557 DOI: 10.2174/1566523223666221118160932] [Citation(s) in RCA: 3] [Impact Index Per Article: 3.0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 05/05/2022] [Revised: 09/27/2022] [Accepted: 10/11/2022] [Indexed: 11/23/2022]
22
Szwec S, Kapłucha Z, Chamberlain JS, Konieczny P. Dystrophin- and Utrophin-Based Therapeutic Approaches for Treatment of Duchenne Muscular Dystrophy: A Comparative Review. BioDrugs 2024;38:95-119. [PMID: 37917377 PMCID: PMC10789850 DOI: 10.1007/s40259-023-00632-3] [Citation(s) in RCA: 3] [Impact Index Per Article: 3.0] [Reference Citation Analysis] [Abstract] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Accepted: 10/10/2023] [Indexed: 11/04/2023]
23
Punetha M, Saini S, Chaudhary S, Yadav PS, Whitworth K, Green J, Kumar D, Kues WA. Induced Pluripotent Stem Cells in the Era of Precise Genome Editing. Curr Stem Cell Res Ther 2024;19:307-315. [PMID: 36880183 DOI: 10.2174/1574888x18666230307115326] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 07/14/2022] [Revised: 11/22/2022] [Accepted: 12/06/2022] [Indexed: 03/08/2023]
24
Lotfi M, Morshedi Rad D, Mashhadi SS, Ashouri A, Mojarrad M, Mozaffari-Jovin S, Farrokhi S, Hashemi M, Lotfi M, Ebrahimi Warkiani M, Abbaszadegan MR. Recent Advances in CRISPR/Cas9 Delivery Approaches for Therapeutic Gene Editing of Stem Cells. Stem Cell Rev Rep 2023;19:2576-2596. [PMID: 37723364 PMCID: PMC10661828 DOI: 10.1007/s12015-023-10585-3] [Citation(s) in RCA: 14] [Impact Index Per Article: 7.0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Accepted: 06/30/2023] [Indexed: 09/20/2023]
25
Hosokawa M, Mikawa R, Hagiwara A, Okuno Y, Awaya T, Yamamoto Y, Takahashi S, Yamaki H, Osawa M, Setoguchi Y, Saito MK, Abe S, Hirai T, Gotoh S, Hagiwara M. Cryptotanshinone is a candidate therapeutic agent for interstitial lung disease associated with a BRICHOS-domain mutation of SFTPC. iScience 2023;26:107731. [PMID: 37701577 PMCID: PMC10494175 DOI: 10.1016/j.isci.2023.107731] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 12/19/2022] [Revised: 08/05/2023] [Accepted: 08/23/2023] [Indexed: 09/14/2023]  Open
26
Molugu K, Khajanchi N, Lazzarotto CR, Tsai SQ, Saha K. Trichostatin A for Efficient CRISPR-Cas9 Gene Editing of Human Pluripotent Stem Cells. CRISPR J 2023;6:473-485. [PMID: 37676985 PMCID: PMC10611976 DOI: 10.1089/crispr.2023.0033] [Citation(s) in RCA: 1] [Impact Index Per Article: 0.5] [Reference Citation Analysis] [Abstract] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 05/27/2023] [Accepted: 07/31/2023] [Indexed: 09/09/2023]  Open
27
Shintani T, Imamura C, Ueyama-Toba Y, Inui J, Watanabe A, Mizuguchi H. Establishment of UGT1A1-knockout human iPS-derived hepatic organoids for UGT1A1-specific kinetics and toxicity evaluation. Mol Ther Methods Clin Dev 2023;30:429-442. [PMID: 37663646 PMCID: PMC10471830 DOI: 10.1016/j.omtm.2023.08.003] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 01/24/2023] [Accepted: 08/08/2023] [Indexed: 09/05/2023]
28
Kita Y, Okuzaki Y, Naoe Y, Lee J, Bang U, Okawa N, Ichiki A, Jonouchi T, Sakurai H, Kojima Y, Hotta A. Dual CRISPR-Cas3 system for inducing multi-exon skipping in DMD patient-derived iPSCs. Stem Cell Reports 2023;18:1753-1765. [PMID: 37625413 PMCID: PMC10545483 DOI: 10.1016/j.stemcr.2023.07.007] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 10/03/2022] [Revised: 07/30/2023] [Accepted: 07/31/2023] [Indexed: 08/27/2023]  Open
29
Gapinske M, Winter J, Swami D, Gapinske L, Woods WS, Shirguppe S, Miskalis A, Busza A, Joulani D, Kao CJ, Kostan K, Bigot A, Bashir R, Perez-Pinera P. Targeting Duchenne muscular dystrophy by skipping DMD exon 45 with base editors. MOLECULAR THERAPY. NUCLEIC ACIDS 2023;33:572-586. [PMID: 37637209 PMCID: PMC10448430 DOI: 10.1016/j.omtn.2023.07.029] [Citation(s) in RCA: 5] [Impact Index Per Article: 2.5] [Reference Citation Analysis] [Abstract] [Key Words] [Grants] [Track Full Text] [Download PDF] [Figures] [Subscribe] [Scholar Register] [Received: 12/15/2022] [Accepted: 07/25/2023] [Indexed: 08/29/2023]
30
Bez Batti Angulski A, Hosny N, Cohen H, Martin AA, Hahn D, Bauer J, Metzger JM. Duchenne muscular dystrophy: disease mechanism and therapeutic strategies. Front Physiol 2023;14:1183101. [PMID: 37435300 PMCID: PMC10330733 DOI: 10.3389/fphys.2023.1183101] [Citation(s) in RCA: 56] [Impact Index Per Article: 28.0] [Reference Citation Analysis] [Abstract] [Key Words] [Grants] [Track Full Text] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 03/09/2023] [Accepted: 05/24/2023] [Indexed: 07/13/2023]  Open
31
Lučanský V, Holubeková V, Kolková Z, Halašová E, Samec M, Golubnitschaja O. Multi-faceted CRISPR/Cas technological innovation aspects in the framework of 3P medicine. EPMA J 2023;14:201-217. [PMID: 37275547 PMCID: PMC10201107 DOI: 10.1007/s13167-023-00324-6] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Grants] [Track Full Text] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 05/02/2023] [Accepted: 05/10/2023] [Indexed: 06/07/2023]
32
Eslahi A, Alizadeh F, Avan A, Ferns GA, Moghbeli M, Reza Abbaszadegan M, Mojarrad M. New advancements in CRISPR based gene therapy of Duchenne muscular dystrophy. Gene 2023;867:147358. [PMID: 36914142 DOI: 10.1016/j.gene.2023.147358] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 11/25/2022] [Revised: 03/02/2023] [Accepted: 03/07/2023] [Indexed: 03/13/2023]
33
Muto V, Benigni F, Magliocca V, Borghi R, Flex E, Pallottini V, Rosa A, Compagnucci C, Tartaglia M. CRISPR/Cas9 and piggyBac Transposon-Based Conversion of a Pathogenic Biallelic TBCD Variant in a Patient-Derived iPSC Line Allows Correction of PEBAT-Related Endophenotypes. Int J Mol Sci 2023;24:ijms24097988. [PMID: 37175696 PMCID: PMC10178052 DOI: 10.3390/ijms24097988] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 03/07/2023] [Revised: 04/20/2023] [Accepted: 04/21/2023] [Indexed: 05/15/2023]  Open
34
Wang P, Li H, Zhu M, Han RY, Guo S, Han R. Correction of DMD in human iPSC-derived cardiomyocytes by base-editing-induced exon skipping. Mol Ther Methods Clin Dev 2023;28:40-50. [PMID: 36588820 PMCID: PMC9792405 DOI: 10.1016/j.omtm.2022.11.010] [Citation(s) in RCA: 14] [Impact Index Per Article: 7.0] [Reference Citation Analysis] [Abstract] [Key Words] [Grants] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 12/13/2021] [Accepted: 11/29/2022] [Indexed: 12/03/2022]
35
Padmaswari MH, Agrawal S, Jia MS, Ivy A, Maxenberger DA, Burcham LA, Nelson CE. Delivery challenges for CRISPR-Cas9 genome editing for Duchenne muscular dystrophy. BIOPHYSICS REVIEWS 2023;4:011307. [PMID: 36864908 PMCID: PMC9969352 DOI: 10.1063/5.0131452] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Grants] [Track Full Text] [Subscribe] [Scholar Register] [Received: 10/20/2022] [Accepted: 01/19/2023] [Indexed: 06/18/2023]
36
Sakuma T, Yamamoto T. Updated Overview of TALEN Construction Systems. Methods Mol Biol 2023;2637:27-39. [PMID: 36773135 DOI: 10.1007/978-1-0716-3016-7_2] [Citation(s) in RCA: 3] [Impact Index Per Article: 1.5] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 02/12/2023]
37
Kawada R, Jonouchi T, Kagita A, Sato M, Hotta A, Sakurai H. Establishment of quantitative and consistent in vitro skeletal muscle pathological models of myotonic dystrophy type 1 using patient-derived iPSCs. Sci Rep 2023;13:94. [PMID: 36631509 PMCID: PMC9834395 DOI: 10.1038/s41598-022-26614-z] [Citation(s) in RCA: 3] [Impact Index Per Article: 1.5] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 08/03/2022] [Accepted: 12/16/2022] [Indexed: 01/13/2023]  Open
38
Milanese JS, Marcotte R, Costain WJ, Kablar B, Drouin S. Roles of Skeletal Muscle in Development: A Bioinformatics and Systems Biology Overview. ADVANCES IN ANATOMY, EMBRYOLOGY, AND CELL BIOLOGY 2023;236:21-55. [PMID: 37955770 DOI: 10.1007/978-3-031-38215-4_2] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Subscribe] [Scholar Register] [Indexed: 11/14/2023]
39
Aslan A, Yuka SA. Stem Cell-Based Therapeutic Approaches in Genetic Diseases. ADVANCES IN EXPERIMENTAL MEDICINE AND BIOLOGY 2023;1436:19-53. [PMID: 36735185 DOI: 10.1007/5584_2023_761] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Subscribe] [Scholar Register] [Indexed: 02/04/2023]
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Caron L, Testa S, Magdinier F. Induced Pluripotent Stem Cells for Modeling Physiological and Pathological Striated Muscle Complexity. J Neuromuscul Dis 2023;10:761-776. [PMID: 37522215 PMCID: PMC10578229 DOI: 10.3233/jnd-230076] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Accepted: 07/13/2023] [Indexed: 08/01/2023]
41
Nalbandian M, Zhao M, Sakurai H. Evaluation of hiPSC-Derived Muscle Progenitor Cell Transplantation in a Mouse Duchenne Muscular Dystrophy Model. Methods Mol Biol 2023;2587:527-536. [PMID: 36401048 DOI: 10.1007/978-1-0716-2772-3_28] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 06/16/2023]
42
Berling E, Nicolle R, Laforêt P, Ronzitti G. Gene therapy review: Duchenne muscular dystrophy case study. Rev Neurol (Paris) 2023;179:90-105. [PMID: 36517287 DOI: 10.1016/j.neurol.2022.11.005] [Citation(s) in RCA: 1] [Impact Index Per Article: 0.5] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 10/27/2022] [Accepted: 11/17/2022] [Indexed: 12/14/2022]
43
Wani AK, Akhtar N, Singh R, Prakash A, Raza SHA, Cavalu S, Chopra C, Madkour M, Elolimy A, Hashem NM. Genome centric engineering using ZFNs, TALENs and CRISPR-Cas9 systems for trait improvement and disease control in Animals. Vet Res Commun 2023;47:1-16. [PMID: 35781172 DOI: 10.1007/s11259-022-09967-8] [Citation(s) in RCA: 8] [Impact Index Per Article: 4.0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 04/26/2022] [Accepted: 06/24/2022] [Indexed: 01/27/2023]
44
Advances in Gene Therapy Techniques to Treat LRRK2 Gene Mutation. Biomolecules 2022;12:biom12121814. [PMID: 36551242 PMCID: PMC9775085 DOI: 10.3390/biom12121814] [Citation(s) in RCA: 2] [Impact Index Per Article: 0.7] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 11/21/2022] [Revised: 12/02/2022] [Accepted: 12/02/2022] [Indexed: 12/12/2022]  Open
45
Preparation of NanoMEDIC Extracellular Vesicles to Deliver CRISPR-Cas9 Ribonucleoproteins for Genomic Exon Skipping. Methods Mol Biol 2022;2587:427-453. [PMID: 36401042 DOI: 10.1007/978-1-0716-2772-3_22] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 11/19/2022]
46
Dong M, Liu J, Liu C, Wang H, Sun W, Liu B. CRISPR/CAS9: A promising approach for the research and treatment of cardiovascular diseases. Pharmacol Res 2022;185:106480. [PMID: 36191879 DOI: 10.1016/j.phrs.2022.106480] [Citation(s) in RCA: 10] [Impact Index Per Article: 3.3] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Scholar Register] [Received: 07/02/2022] [Revised: 09/27/2022] [Accepted: 09/29/2022] [Indexed: 10/31/2022]
47
CRISPR-Based Therapeutic Gene Editing for Duchenne Muscular Dystrophy: Advances, Challenges and Perspectives. Cells 2022;11:cells11192964. [PMID: 36230926 PMCID: PMC9564082 DOI: 10.3390/cells11192964] [Citation(s) in RCA: 12] [Impact Index Per Article: 4.0] [Reference Citation Analysis] [Abstract] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 08/11/2022] [Revised: 09/06/2022] [Accepted: 09/09/2022] [Indexed: 11/19/2022]  Open
48
Nishitani-Isa M, Mukai K, Honda Y, Nihira H, Tanaka T, Shibata H, Kodama K, Hiejima E, Izawa K, Kawasaki Y, Osawa M, Katata Y, Onodera S, Watanabe T, Uchida T, Kure S, Takita J, Ohara O, Saito MK, Nishikomori R, Taguchi T, Sasahara Y, Yasumi T. Trapping of CDC42 C-terminal variants in the Golgi drives pyrin inflammasome hyperactivation. J Exp Med 2022;219:213184. [PMID: 35482294 PMCID: PMC9059393 DOI: 10.1084/jem.20211889] [Citation(s) in RCA: 26] [Impact Index Per Article: 8.7] [Reference Citation Analysis] [Abstract] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 09/06/2021] [Revised: 02/28/2022] [Accepted: 03/31/2022] [Indexed: 12/12/2022]  Open
49
Morimoto Y, Tokumitsu A, Sone T, Hirota Y, Tamura R, Sakamoto A, Nakajima K, Toda M, Kawakami Y, Okano H, Ohta S. TPT1 Supports Proliferation of Neural Stem/Progenitor Cells and Brain Tumor Initiating Cells Regulated by Macrophage Migration Inhibitory Factor (MIF). Neurochem Res 2022;47:2741-2756. [PMID: 35622214 DOI: 10.1007/s11064-022-03629-6] [Citation(s) in RCA: 1] [Impact Index Per Article: 0.3] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 01/28/2022] [Revised: 05/03/2022] [Accepted: 05/05/2022] [Indexed: 11/25/2022]
50
Long-Term Protective Effect of Human Dystrophin Expressing Chimeric (DEC) Cell Therapy on Amelioration of Function of Cardiac, Respiratory and Skeletal Muscles in Duchenne Muscular Dystrophy. Stem Cell Rev Rep 2022;18:2872-2892. [PMID: 35590083 PMCID: PMC9622520 DOI: 10.1007/s12015-022-10384-2] [Citation(s) in RCA: 10] [Impact Index Per Article: 3.3] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Accepted: 04/26/2022] [Indexed: 12/12/2022]
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