DOAJ Open Access 2017

CRISPR/Cas9-mediated targeted gene correction in amyotrophic lateral sclerosis patient iPSCs

Lixia Wang Fei Yi Lina Fu Jiping Yang Si Wang +11 lainnya

Abstrak

Abstract Amyotrophic lateral sclerosis (ALS) is a complex neurodegenerative disease with cellular and molecular mechanisms yet to be fully described. Mutations in a number of genes including SOD1 and FUS are associated with familial ALS. Here we report the generation of induced pluripotent stem cells (iPSCs) from fibroblasts of familial ALS patients bearing SOD1 +/A272C and FUS +/G1566A mutations, respectively. We further generated gene corrected ALS iPSCs using CRISPR/Cas9 system. Genome-wide RNA sequencing (RNA-seq) analysis of motor neurons derived from SOD1 +/A272C and corrected iPSCs revealed 899 aberrant transcripts. Our work may shed light on discovery of early biomarkers and pathways dysregulated in ALS, as well as provide a basis for novel therapeutic strategies to treat ALS.

Topik & Kata Kunci

Cytology Animal biochemistry

Penulis (16)

Lixia Wang

Fei Yi

Lina Fu

Jiping Yang

Si Wang

Zhaoxia Wang

Keiichiro Suzuki

Liang Sun

Xiuling Xu

Yang Yu

Jie Qiao

Juan Carlos Izpisua Belmonte

Ze Yang

Yun Yuan

Jing Qu

Guang-Hui Liu

Format Sitasi

APA MLA BibTeX

Wang, L., Yi, F., Fu, L., Yang, J., Wang, S., Wang, Z. et al. (2017). CRISPR/Cas9-mediated targeted gene correction in amyotrophic lateral sclerosis patient iPSCs. https://doi.org/10.1007/s13238-017-0397-3

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Informasi Jurnal

Tahun Terbit: 2017
Sumber Database: DOAJ
DOI: 10.1007/s13238-017-0397-3
Akses: Open Access ✓