Cellular Reprogramming, Genome Editing, and Alternative CRISPR Cas9 Technologies for Precise Gene Therapy of Duchenne Muscular Dystrophy
Joint Authors
Gee, Peter
Xu, Huaigeng
Hotta, Akitsu
Source
Issue
Vol. 2017, Issue 2017 (31 Dec. 2017), pp.1-11, 11 p.
Publisher
Hindawi Publishing Corporation
Publication Date
2017-05-18
Country of Publication
Egypt
No. of Pages
11
Abstract EN
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 muscular dystrophy (DMD) has been an exemplary monogenic disease model for combining these technologies to demonstrate that genome editing can correct genetic mutations in DMD patient-derived iPSCs.
DMD is an X-linked genetic disorder caused by mutations that disrupt the open reading frame of the dystrophin gene, which plays a critical role in stabilizing muscle cells during contraction and relaxation.
The CRISPR Cas9 system has been shown to be capable of targeting the dystrophin gene and rescuing its expression in in vitro patient-derived iPSCs and in vivo DMD mouse models.
In this review, we highlight recent advances made using the CRISPR Cas9 system to correct genetic mutations and discuss how emerging CRISPR technologies and iPSCs in a combined platform can play a role in bringing a therapy for DMD closer to the clinic.
American Psychological Association (APA)
Gee, Peter& Xu, Huaigeng& Hotta, Akitsu. 2017. Cellular Reprogramming, Genome Editing, and Alternative CRISPR Cas9 Technologies for Precise Gene Therapy of Duchenne Muscular Dystrophy. Stem Cells International،Vol. 2017, no. 2017, pp.1-11.
https://search.emarefa.net/detail/BIM-1202307
Modern Language Association (MLA)
Gee, Peter…[et al.]. Cellular Reprogramming, Genome Editing, and Alternative CRISPR Cas9 Technologies for Precise Gene Therapy of Duchenne Muscular Dystrophy. Stem Cells International No. 2017 (2017), pp.1-11.
https://search.emarefa.net/detail/BIM-1202307
American Medical Association (AMA)
Gee, Peter& Xu, Huaigeng& Hotta, Akitsu. Cellular Reprogramming, Genome Editing, and Alternative CRISPR Cas9 Technologies for Precise Gene Therapy of Duchenne Muscular Dystrophy. Stem Cells International. 2017. Vol. 2017, no. 2017, pp.1-11.
https://search.emarefa.net/detail/BIM-1202307
Data Type
Journal Articles
Language
English
Notes
Includes bibliographical references
Record ID
BIM-1202307