Article

Protein Assembly Modulation: A New Approach to ALS Therapeutics

2023-07-26

Abstract excerpt

Amyotrophic Lateral Sclerosis (ALS) is a neurodegenerative disease with a complex, multifactorial pathophysiology, most commonly manifest as loss of motor neurons. We introduce a new mechanism of ALS pathogenesis via a novel drug-like small molecule series that targets protein disulfide isomerase (PDI) within a previously unappreciated transient and energy-dependent multi-protein complex. This novel drug was found...

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Literature Corpus work: 7013120d-db16-5f4a-8ff0-a3123e83e1f0
DOI: 10.1101/2023.07.24.550252

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