Author

F. Ann Ran

Broad Institute

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Neuroscience· 1

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Approach:experimental assay· 1 Organism:mouse· 1

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Ana Rita Fernandes· 1 shared Ana Vasconcelos‐FerreiraiD· 1 shared Carlos A. MatosiD· 1 shared Clévio NóbregaiD· 1 shared Daniela Camargo de OliveiraiD· 1 shared Dina PereiraiD· 1 shared Feng ZhangiD· 1 shared Le CongiD· 1 shared

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1 shared paperNeuroscience

Ana Rita Fernandes

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Recent·Most cited

NeurosciencebioRxivPreprintFeb 14, 2025

Gene Editing for ATXN3 Inactivation in Machado-Joseph disease: CRISPR-Cas9 as a Therapeutic Alternative to TALEN-Induced Toxicity

Sara Monteiro Lopes, Miguel Monteiro Lopes, Daniela Camargo de Oliveira…·Luís Pereira de Almeida

This study compares CRISPR-Cas9 and TALEN gene editing in a mouse model of Machado-Joseph disease, showing CRISPR-Cas9 reduces ATXN3 aggregates and delays disease progression.

DOI 10.1101/2025.02.14.637261

mouseexperimental assay

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