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

• Main Authors: Peter Gee (Author), Huaigeng Xu (Author), Akitsu Hotta (Author)
• Formato: Livro
• Publicado em: Hindawi Limited, 2017-01-01T00:00:00Z.
• Assuntos: article
• Acesso em linha: Connect to this object online.

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...