CRISPR expands insight into the mechanisms of ALS and FTD

Sarah Pickles, Leonard Petrucelli

Research output: Contribution to journalArticle

Abstract

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
DOIs
StateAccepted/In press - Apr 25 2018

Fingerprint

Clustered Regularly Interspaced Short Palindromic Repeats
Hand Strength
Therapeutics
Frontotemporal Dementia With Motor Neuron Disease
Gene Editing

ASJC Scopus subject areas

  • Clinical Neurology
  • Cellular and Molecular Neuroscience

Cite this

CRISPR expands insight into the mechanisms of ALS and FTD. / Pickles, Sarah; Petrucelli, Leonard.

In: Nature Reviews Neurology, 25.04.2018, p. 1-2.

Research output: Contribution to journalArticle

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