Cargando…

Therapeutic Genome Editing for Myotonic Dystrophy Type 1 Using CRISPR/Cas9

Myotonic dystrophy type 1 (DM1) is caused by a CTG nucleotide repeat expansion within the 3′ UTR of the Dystrophia Myotonica protein kinase gene. In this study, we explored therapeutic genome editing using CRISPR/Cas9 via targeted deletion of expanded CTG repeats and targeted insertion of polyadenyl...

Descripción completa

Detalles Bibliográficos
Autores principales:	Wang, Yanlin, Hao, Lei, Wang, Hongcai, Santostefano, Katherine, Thapa, Arjun, Cleary, John, Li, Hui, Guo, Xiuming, Terada, Naohiro, Ashizawa, Tetsuo, Xia, Guangbin
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2018
Materias:	Original Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6225032/ https://www.ncbi.nlm.nih.gov/pubmed/30274788 http://dx.doi.org/10.1016/j.ymthe.2018.09.003

Ejemplares similares

A CRISPR-Cas13a Based Strategy That Tracks and Degrades Toxic RNA in Myotonic Dystrophy Type 1
por: Zhang, Nan, et al.
Publicado: (2020)

Human iPSC Models to Study Orphan Diseases: Muscular Dystrophies
por: Xia, Guangbin, et al.
Publicado: (2018)

CRISPR/Cas Applications in Myotonic Dystrophy: Expanding Opportunities
por: Raaijmakers, Renée H.L., et al.
Publicado: (2019)

Efficient CRISPR/Cas9-mediated editing of trinucleotide repeat expansion in myotonic dystrophy patient-derived iPS and myogenic cells
por: Dastidar, Sumitava, et al.
Publicado: (2018)

CRISPR/Cas9-Induced (CTG⋅CAG)(n) Repeat Instability in the Myotonic Dystrophy Type 1 Locus: Implications for Therapeutic Genome Editing
por: van Agtmaal, Ellen L., et al.
Publicado: (2017)

Unexpected Mutations by CRISPR-Cas9 CTG Repeat Excision in Myotonic Dystrophy and Use of CRISPR Interference as an Alternative Approach
por: Ikeda, Miki, et al.
Publicado: (2020)

CRISPR/Cas9 knockout of USP18 enhances type I IFN responsiveness and restricts HIV‐1 infection in macrophages
por: Taylor, Jared P., et al.
Publicado: (2018)

Comprehensive transcriptome-wide analysis of spliceopathy correction of myotonic dystrophy using CRISPR-Cas9 in iPSCs-derived cardiomyocytes
por: Dastidar, Sumitava, et al.
Publicado: (2022)

Myotonic dystrophy
por: Patterson, VH
Publicado: (1990)

CRISPR/Cas9-Mediated Deletion of CTG Expansions Recovers Normal Phenotype in Myogenic Cells Derived from Myotonic Dystrophy 1 Patients
por: Provenzano, Claudia, et al.
Publicado: (2017)

Cancer and Myotonic Dystrophy
por: D’Ambrosio, Eleonora S., et al.
Publicado: (2023)

Consensus-based care recommendations for congenital and childhood-onset myotonic dystrophy type 1
por: Johnson, Nicholas E., et al.
Publicado: (2019)

Delivery strategies for CRISPR/Cas genome editing tool for retinal dystrophies: challenges and opportunities
por: Lohia, Aayushi, et al.
Publicado: (2022)

Mitigating RNA Toxicity in Myotonic Dystrophy using Small Molecules
por: Reddy, Kaalak, et al.
Publicado: (2019)

CRISPR-Cas9 DNA Base-Editing and Prime-Editing
por: Kantor, Ariel, et al.
Publicado: (2020)

CRISPR/Cas9 or prime editing? – It depends on…
por: Schenke, Dirk
Publicado: (2020)

Multiplex CRISPR/Cas9-Based Genome Editing for Correction of Dystrophin Mutations that Cause Duchenne Muscular Dystrophy
por: Ousterout, David G., et al.
Publicado: (2015)

Promising therapeutic approaches using CRISPR/Cas9 genome editing technology in the treatment of Duchenne muscular dystrophy
por: Mollanoori, Hasan, et al.
Publicado: (2020)

CRISPR-Cas9-Based Genome Editing and Cytidine Base Editing in Acinetobacter baumannii
por: Wang, Yu, et al.
Publicado: (2020)

Strategies in the delivery of Cas9 ribonucleoprotein for CRISPR/Cas9 genome editing
por: Zhang, Song, et al.
Publicado: (2021)

Desflurane anaesthesia in myotonic dystrophy
por: Gandhi, Ranju, et al.
Publicado: (2011)

Myotonic Dystrophy: An Anaesthetic Dilemma
por: Gupta, N, et al.
Publicado: (2009)

Cardiac involvement in myotonic dystrophy
por: Khalighi, Koroush, et al.
Publicado: (2015)

Steakhouse Syndrome in Myotonic Dystrophy
por: Ogasawara, Nobuhiko, et al.
Publicado: (2017)

Cutaneous findings in myotonic dystrophy
por: Kong, Ha Eun, et al.
Publicado: (2022)

CRISPR/Cas9-mediated genome editing in nonhuman primates
por: Kang, Yu, et al.
Publicado: (2019)

Molecular Therapies for Myotonic Dystrophy Type 1: From Small Drugs to Gene Editing
por: Izzo, Mariapaola, et al.
Publicado: (2022)

Application of CRISPR/Cas9 Nuclease in Amphioxus Genome Editing
por: Su, Liuru, et al.
Publicado: (2020)

Applications of CRISPR/Cas9 for the Treatment of Duchenne Muscular Dystrophy
por: Lim, Kenji Rowel Q., et al.
Publicado: (2018)

Cellular Reprogramming, Genome Editing, and Alternative CRISPR Cas9 Technologies for Precise Gene Therapy of Duchenne Muscular Dystrophy
por: Gee, Peter, et al.
Publicado: (2017)

Duchenne muscular dystrophy cell culture models created by CRISPR/Cas9 gene editing and their application in drug screening
por: Soblechero-Martín, Patricia, et al.
Publicado: (2021)

Inducible in vivo genome editing with CRISPR/Cas9
por: Dow, Lukas E, et al.
Publicado: (2015)

Efficient Mitochondrial Genome Editing by CRISPR/Cas9
por: Jo, Areum, et al.
Publicado: (2015)

CRISPR/Cas9: Transcending the Reality of Genome Editing
por: Chira, Sergiu, et al.
Publicado: (2017)

Genome Editing in Cotton with the CRISPR/Cas9 System
por: Gao, Wei, et al.
Publicado: (2017)

CRISPR/Cas9 Editing for Gaucher Disease Modelling
por: Pavan, Eleonora, et al.
Publicado: (2020)

Spatiotemporal control of CRISPR/Cas9 gene editing
por: Zhuo, Chenya, et al.
Publicado: (2021)

Nanoparticle Delivery of CRISPR/Cas9 for Genome Editing
por: Duan, Li, et al.
Publicado: (2021)

CRISPR–Cas9 Based Bacteriophage Genome Editing
por: Zhang, Xueli, et al.
Publicado: (2022)

CRISPR/Cas9 genome editing for neurodegenerative diseases
por: Nouri Nojadeh, Jafar, et al.
Publicado: (2023)

Cannot write session to /tmp/vufind_sessions/sess_li6kgpu9ma1oior47pgnjcoubl