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Article

The HIV protease inhibitor, ritonavir, corrects diverse brain phenotypes across development in mouse model of DYT-TOR1A dystonia.

Science translational medicine - 18 Aug 2021

Caffall Zachary F, Wilkes Bradley J, Hernández-Martinez Ricardo, Rittiner Joseph E, Fox Jennifer T, Wan Kanny K, Shipman Miranda K, Titus Steven A, Zhang Ya-Qin, Patnaik Samarjit, Hall Matthew D, Boxer Matthew B, Shen Min, Li Zhuyin, Vaillancourt David E, Calakos Nicole

Abstract excerpt

Dystonias are a group of chronic movement-disabling disorders for which highly effective oral medications or disease-modifying therapies are lacking. The most effective treatments require invasive procedures such as deep brain stimulation. In this study, we used a high-throughput assay based on a monogenic form of dystonia, DYT1 (DYT-TOR1A), to screen a library of compounds approved for use in humans, the NCATS...

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Identifiers

PMID: 34408078
DOI: 10.1126/scitranslmed.abd3904
PMCID: PMC8827175

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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