Cargando…

Cellular Reprogramming, Genome Editing, and Alternative CRISPR Cas9 Technologies for Precise Gene Therapy of Duchenne Muscular Dystrophy

In the past decade, the development of two innovative technologies, namely, induced pluripotent stem cells (iPSCs) and the CRISPR Cas9 system, has enabled researchers to model diseases derived from patient cells and precisely edit DNA sequences of interest, respectively. In particular, Duchenne musc...

Descripción completa

Detalles Bibliográficos
Autores principales:	Gee, Peter, Xu, Huaigeng, Hotta, Akitsu
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Hindawi 2017
Materias:	Review Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5451761/ https://www.ncbi.nlm.nih.gov/pubmed/28607562 http://dx.doi.org/10.1155/2017/8765154

Ejemplares similares

Genome Editing Gene Therapy for Duchenne Muscular Dystrophy
por: Hotta, Akitsu
Publicado: (2015)

Precise Correction of the Dystrophin Gene in Duchenne Muscular Dystrophy Patient Induced Pluripotent Stem Cells by TALEN and CRISPR-Cas9
por: Li, Hongmei Lisa, et al.
Publicado: (2014)

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

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

CRISPR technologies for the treatment of Duchenne muscular dystrophy
por: Choi, Eunyoung, et al.
Publicado: (2021)

Optimized electroporation of CRISPR-Cas9/gRNA ribonucleoprotein complex for selection-free homologous recombination in human pluripotent stem cells
por: Xu, Huaigeng, et al.
Publicado: (2021)

CRISPR Therapeutics for Duchenne Muscular Dystrophy
por: Erkut, Esra, et al.
Publicado: (2022)

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

CRISPR-Cas3 induces broad and unidirectional genome editing in human cells
por: Morisaka, Hiroyuki, et al.
Publicado: (2019)

Long-term Evaluation of AAV-CRISPR Genome Editing for Duchenne Muscular Dystrophy
por: Nelson, Christopher E., et al.
Publicado: (2019)

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)

CRISPR-Based Therapeutic Gene Editing for Duchenne Muscular Dystrophy: Advances, Challenges and Perspectives
por: Chen, Guofang, et al.
Publicado: (2022)

A novel rabbit model of Duchenne muscular dystrophy generated by CRISPR/Cas9
por: Sui, Tingting, et al.
Publicado: (2018)

Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy
por: Bengtsson, Niclas E., et al.
Publicado: (2017)

A humanized knockin mouse model of Duchenne muscular dystrophy and its correction by CRISPR-Cas9 therapeutic gene editing
por: Zhang, Yu, et al.
Publicado: (2022)

Precise correction of Duchenne muscular dystrophy exon deletion mutations by base and prime editing
por: Chemello, F., et al.
Publicado: (2021)

CRISPR-Generated Animal Models of Duchenne Muscular Dystrophy
por: Lim, Kenji Rowel Q., et al.
Publicado: (2020)

Efficient ssODN-Mediated Targeting by Avoiding Cellular Inhibitory RNAs through Precomplexed CRISPR-Cas9/sgRNA Ribonucleoprotein
por: Kagita, Akihiro, et al.
Publicado: (2021)

CRISPR-Cas9-mediated exon skipping as a cardioprotective strategy in Duchenne muscular dystrophy
por: Wilton-Clark, Harry, et al.
Publicado: (2023)

Corrigendum: Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy
por: Bengtsson, Niclas E., et al.
Publicado: (2017)

Site-specific randomization of the endogenous genome by a regulatable CRISPR-Cas9 piggyBac system in human cells
por: Ishida, Kentaro, et al.
Publicado: (2018)

Precision Medicine and Exercise Therapy in Duchenne Muscular Dystrophy
por: Kostek, Matthew
Publicado: (2019)

CRISPR/Cas9 editing of directly reprogrammed myogenic progenitors restores dystrophin expression in a mouse model of muscular dystrophy
por: Domenig, Seraina A., et al.
Publicado: (2022)

Challenges associated with homologous directed repair using CRISPR-Cas9 and TALEN to edit the DMD genetic mutation in canine Duchenne muscular dystrophy
por: Mata López, Sara, et al.
Publicado: (2020)

Genome editing for Duchenne muscular dystrophy: a glimpse of the future?
por: Kupatt, Christian, et al.
Publicado: (2021)

Cellular senescence-mediated exacerbation of Duchenne muscular dystrophy
por: Sugihara, Hidetoshi, et al.
Publicado: (2020)

Correction: Challenges associated with homologous directed repair using CRISPR-Cas9 and TALEN to edit the DMD genetic mutation in canine Duchenne muscular dystrophy
por: Mata López, Sara, et al.
Publicado: (2020)

CRISPR-Cas9 corrects Duchenne muscular dystrophy exon 44 deletion mutations in mice and human cells
por: Min, Yi-Li, et al.
Publicado: (2019)

Single-swap editing for the correction of common Duchenne muscular dystrophy mutations
por: Chai, Andreas C., et al.
Publicado: (2023)

A muscle fatigue-like contractile decline was recapitulated using skeletal myotubes from Duchenne muscular dystrophy patient-derived iPSCs
por: Uchimura, Tomoya, et al.
Publicado: (2021)

Alternative instrument for the evaluation of handgrip strength in Duchenne muscular dystrophy
por: de Souza, Mariana Angélica, et al.
Publicado: (2022)

A medium-chain triglyceride containing ketogenic diet exacerbates cardiomyopathy in a CRISPR/Cas9 gene-edited rat model with Duchenne muscular dystrophy
por: Fujikura, Yuri, et al.
Publicado: (2022)

Survival in Duchenne muscular dystrophy
por: RALL, SUSANNE, et al.
Publicado: (2012)

Theragnosis for Duchenne Muscular Dystrophy
por: Luce, Leonela, et al.
Publicado: (2021)

Enhanced CRISPR-Cas9 correction of Duchenne muscular dystrophy in mice by a self-complementary AAV delivery system
por: Zhang, Yu, et al.
Publicado: (2020)

Emerging gene editing strategies for Duchenne muscular dystrophy targeting stem cells
por: Bertoni, Carmen
Publicado: (2014)

Genome Editing-Mediated Utrophin Upregulation in Duchenne Muscular Dystrophy Stem Cells
por: Sengupta, Kasturi, et al.
Publicado: (2020)

CRISPR applications for Duchenne muscular dystrophy: From animal models to potential therapies
por: Chey, Yu C. J., et al.
Publicado: (2022)

Contractile Activity of Myotubes Derived from Human Induced Pluripotent Stem Cells: A Model of Duchenne Muscular Dystrophy
por: Yoshioka, Kantaro, et al.
Publicado: (2021)

Hypokalemia complicating Duchenne muscular dystrophy.
por: McDonald, B., et al.
Publicado: (1987)

Cannot write session to /tmp/vufind_sessions/sess_hspobtg756hvbck2ecoi0d1r2s