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Article

CRISPR/Cas9-engineered Drosophila knock-in models to study VCP diseases.

Disease models & mechanisms - 1 Jul 2021

Wall Jordan M, Basu Ankita, Zunica Elizabeth R M, Dubuisson Olga S, Pergola Kathryn, Broussard Joshua P, Kirwan John P, Axelrod Christopher L, Johnson Alyssa E

Abstract excerpt

Mutations in Valosin Containing Protein (VCP) are associated with several degenerative diseases, including multisystem proteinopathy (MSP-1) and amyotrophic lateral sclerosis. However, patients with VCP mutations vary widely in their pathology and clinical penetrance, making it difficult to devise effective treatment strategies. A deeper understanding of how each mutation affects VCP function could enhance the...

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Identifiers

PMID: 34160014
DOI: 10.1242/dmm.048603
PMCID: PMC8325010

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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