Stem Cell Reports
Volume 14, Issue 3, 10 March 2020, Pages 390-405
ArticleKnocking out C9ORF72 Exacerbates Axonal Trafficking Defects Associated with Hexanucleotide Repeat Expansion and Reduces Levels of Heat Shock Proteins
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open access
Keywords
C9ORF72
induced pluripotent stem cells
amyotrophic lateral sclerosis
gene editing
disease modeling
axonal trafficking
heat shock proteins
HSP70
HSP40
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