CRISPR/Cas9-mediated targeted gene correction in amyotrophic lateral sclerosis patient iPSCs
Protein & Cell
;
(12): 365-378, 2017.
Article
in English
| WPRIM
| ID: wpr-756992
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 and FUS 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 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.
Full text:
Available
Index:
WPRIM (Western Pacific)
Main subject:
Therapeutics
/
Genetic Therapy
/
Cell Line
/
Mutation, Missense
/
RNA-Binding Protein FUS
/
Genome-Wide Association Study
/
Induced Pluripotent Stem Cells
/
Clustered Regularly Interspaced Short Palindromic Repeats
/
Superoxide Dismutase-1
/
Genetics
Limits:
Humans
Language:
English
Journal:
Protein & Cell
Year:
2017
Type:
Article
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