RNA-Directed Therapy for C9ORF72-Linked ALS Using Engineered Zinc Finger Nucleases

Abstract

ALS caused by mutations in the gene C9ORF72. GGGGCC hexanucleotide repeat expansions,(G4C2)exp, in the first intron of the C9ORF72 gene is the most common cause of familial ALS (C9-ALS) and have been linked to RNA-mediated pathogenesis by the formation of toxic RNA foci formedfrom both sense (G4C2)exp and antisense (C4G2)exp C9ORF72 transcripts.

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Document Details

Document Type
Technical Report
Publication Date
May 01, 2022
Accession Number
AD1177765

Entities

People

  • Gene W. Yeo

Organizations

  • University of California, San Diego

Tags

Communities of Interest

  • Biomedical

DTIC Thesaurus Topics

  • Antisense Elements (Genetics)
  • Biomedical Research
  • California
  • Carrier Proteins
  • Cell Line
  • Cells
  • Covid-19
  • Department Of Defense
  • Gene Expression
  • Genetic Variation
  • Indirect Costs
  • Medical Personnel
  • Mutations
  • Nervous System
  • Neurodegeneration
  • Neurodegenerative Diseases
  • Neurons
  • Parkinson'S Disease
  • Proteins
  • Sars
  • Stem Cells
  • Therapy

Readers

  • Materials Science and Engineering.
  • Medical Imaging.
  • Molecular Genetics