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Rewiring ALS by modulating the autophagy receptor SQSTM1

Lookup NU author(s): Professor Viktor KorolchukORCiD

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This work is licensed under a Creative Commons Attribution 4.0 International License (CC BY 4.0).

Abstract

© 2026 The Author(s). Drug screening for genetic disorders is limited by difficulty identifying disease-relevant phenotypes. In this issue, Roussange et al., show that reverse phenotypic mapping could uncover therapeutic gene expression signatures. Using this approach, they identified prazosin, which increases SQSTM1 expression and rescues disease phenotypes in iPSC-derived motor neurons and zebrafish model of amyotrophic lateral sclerosis with SQSTM1 haploinsufficiency.

Publication metadata

Author(s): Aubry L, Korolchuk VI, Sarkar S

Publication type: Note

Publication status: Published

Journal: Stem Cell Reports

Year: 2026

Volume: 21

Issue: 7

Print publication date: 14/07/2026

Online publication date: 25/06/2026

Acceptance date: 02/04/2018

ISSN (electronic): 2213-6711

Publisher: Cell Press

URL: https://doi.org/10.1016/j.stemcr.2026.102976

DOI: 10.1016/j.stemcr.2026.102976

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