CRISPR expands insight into the mechanisms of ALS and FTD

Sarah Pickles, Leonard Petrucelli

Research output: Contribution to journalArticle

1 Scopus citations


An incomplete grasp of how the G4C2 repeat expansion in C9orf72 leads to amyotrophic lateral sclerosis and frontotemporal dementia has hindered progress in treatment development. Now, a study has combined unbiased genetic screens and CRISPR–Cas9 gene editing to validate known molecular pathways and identify novel therapeutic targets involved in G4C2 repeat pathogenesis.

Original languageEnglish (US)
Pages (from-to)1-2
Number of pages2
JournalNature Reviews Neurology
StateAccepted/In press - Apr 25 2018

ASJC Scopus subject areas

  • Clinical Neurology
  • Cellular and Molecular Neuroscience

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