Concepedia

Chemical chaperones targeted to the endoplasmic reticulum (ER) and lysosome prevented neurodegeneration in a C9orf72 repeat expansion drosophila amyotrophic lateral sclerosis (ALS) model

Salome Azoulay‐Ginsburg, Michela Di Salvio, Michal Weitman, Michal Afri, Sara S. Ribeiro, Simon Ebbinghaus, Gianluca Cestra, Arie Gruzman

Pharmacological Reports · 2021 · 16 citations · 25 references

DOI

Concepts

References

25

A Hexanucleotide Repeat Expansion in C9ORF72 Is the Cause of Chromosome 9p21-Linked ALS-FTD

Alan E. Renton, Elisa Majounie, Adrian J. Waite et al. · Neuron · 2011 · 4.4K citations · Full text

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NMR Chemical Shifts of Common Laboratory Solvents as Trace Impurities

Hugo E. Gottlieb, Vadim Kotlyar, Abraham Nudelman · The Journal of Organic Chemistry · 1997 · 3.6K citations

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<i>C9orf72</i> repeat expansions cause neurodegeneration in <i>Drosophila</i> through arginine-rich proteins

Sarah Mizielinska, Sebastian Grönke, Teresa Niccoli et al. · Science · 2014 · 735 citations · Full text

Trial of Sodium Phenylbutyrate–Taurursodiol for Amyotrophic Lateral Sclerosis

Sabrina Paganoni, Eric A. Macklin, Suzanne Hendrix et al. · New England Journal of Medicine · 2020 · 505 citations · Full text

In Situ Structure of Neuronal C9orf72 Poly-GA Aggregates Reveals Proteasome Recruitment

Qiang Guo, Carina Lehmer, Antonio Martínez-Sánchez et al. · Cell · 2018 · 410 citations · Full text

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