Induced pluripotent stem cells and genome editing technology as therapeutic strategies for Duchenne muscular dystrophy
https://doi.org/10.19106/JMedSci005301202109
Irwan Saputra Batubara
(1*)
(1) Division of Surgical and Interventional Science, University Colllege London
(*) Corresponding Author
Abstract
Duchenne muscular dystrophy (DMD) is a rare genetic, progressive and devastating skeletal and cardiac muscle disorder due to mutation of the dystrophin gene that affects 1 in 3500 young males. Currently, there is no curative management for this pathology. The development of inducedpluripotent stem cells (iPSCs) offers a promising cell-based strategy for the treatment of muscular dystrophy. Several techniques have been established to generate functional myogenic progenitor cells derived from iPSCs. In addition, technologies in genetic modification using ZFN, TALENs, or CRISPR/Cas9 demonstrate potent methods to restore dystrophin expression. However, current evidence shows that either iPSCs or gene editing carry a risk of oncogenesis caused by the integration of exogenous DNA into the recipient gene. Thus, the safety issue is a major challenge for translating this method into human clinical applications. This review briefly discussed recent developments and progressions of iPSCs as well as genome engineering technologies relevant to regenerative medicine, especially for the treatment of DMD.
Keywords
induced pluripotent; stem cells; Duchenne muscular dystrophy; gene editing; cell-based therapy
DOI:
https://doi.org/10.19106/JMedSci005301202109
Article Metrics
Abstract views : 1490
|
views : 2683
Copyright (c) 2021 Irwan Saputra Batubara
This work is licensed under a
Creative Commons Attribution-NonCommercial 4.0 International License .
piala88 slot/
https://heatbeatmiami.com/
judi bola slot gacor
slot gacor
href="https://whiteroseshub.com/">https://whiteroseshub.com/
mallbet login
mallbet login
mallbet login
<div class="statcounter"><a title="real time web analytics" href="https://statcounter.com/" target="_blank"><img class="statcounter" src="https://c.statcounter.com/10697571/0/23d82b78/0/" alt="real time web analytics"></a></div> <div class="statcounter"><a title="shopify visitor statistics" href="http://statcounter.com/shopify/" target="_blank"><img class="statcounter" src="http://c.statcounter.com/10697571/0/23d82b78/0/" alt="shopify visitor statistics"></a></div>