AAV-based delivery of RNAi targeting ataxin-2 improves survival and pathology in TDP-43 mice

Defne A. Amado; Ashley B. Robbins; Katherine R. Whiteman; Alicia R. Smith; Guillem Chillon; Yonghong Chen; Joshua A. Fuller; Nicholas A. Patty; Aleksandar Izda; Congsheng Cheng; Shareen Nelson; Abigail I. Dichter; Esteban O. Mazzoni; Alex Mas Monteys; Beverly L. Davidson

doi:10.1038/s41467-025-60497-8

AAV-based delivery of RNAi targeting ataxin-2 improves survival and pathology in TDP-43 mice

D Defne A. Amado A Ashley B. Robbins K Katherine R. Whiteman A Alicia R. Smith G Guillem Chillon Y Yonghong Chen (Torch Electron Technology Co., LTD) J Joshua A. Fuller N Nicholas A. Patty A Aleksandar Izda C Congsheng Cheng S Shareen Nelson A Abigail I. Dichter E Esteban O. Mazzoni A Alex Mas Monteys B Beverly L. Davidson (Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children’s Hospital of Philadelphia, University of Pennsylvania)

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Article Details

Journal Nature Communications

Volume / Issue Vol. 16, Issue 1

Published June 25, 2025

DOI 10.1038/s41467-025-60497-8

ISSN 2041-1723

Publisher Nature Portfolio

Journal Info

Nature Communications

Nature Portfolio

ISSN: 2041-1723 Open Access Life Sciences

Authors (15)

D

Defne A. Amado

A

Ashley B. Robbins

K

Katherine R. Whiteman

A

Alicia R. Smith

G

Guillem Chillon

Y

Yonghong Chen

Torch Electron Technology Co., LTD

J

Joshua A. Fuller

N

Nicholas A. Patty

A

Aleksandar Izda

C

Congsheng Cheng

S

Shareen Nelson

A

Abigail I. Dichter

E

Esteban O. Mazzoni

A

Alex Mas Monteys

B

Beverly L. Davidson

Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children’s Hospital of Philadelphia, University of Pennsylvania

AAV-based delivery of RNAi targeting ataxin-2 improves survival and pathology in TDP-43 mice

Article Details

Journal Info

Authors (15)

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