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-GinsburgMichela Di SalvioMichal WeitmanMichal AfriSara RibeiroSimon EbbinghausGianluca CestraArie Gruzman

Published in: Pharmacological reports : PR (2021)

Novel CSs were more effective than 4-PBA; therefore, they might be used as a new class of drug candidates to treat ALS and other protein misfolding disorders.

Keyphrases

amyotrophic lateral sclerosis
endoplasmic reticulum
cancer therapy
fluorescent probe
heat shock
amino acid
binding protein
emergency department
drug induced
heat shock protein