TwinPE delivered as RNA or VLP enables precise deletion of GAA repeats in the FXN gene
Authors:
- Xuntao ZhouXZXuntao Zhou
- Thomas C. TodeschiniTTThomas C. Todeschini
- Tianxiong YuTYTianxiong Yu
- Runyuan WangRWRunyuan Wang
- Bin LiuBLBin Liu
- Annie CollinsACAnnie Collins
- Whitney WelchWWWhitney Welch
- Aditya Valji AnsodariaAAAditya Valji Ansodaria
- Zhiping WengZWZhiping Weng
- Scot A. WolfeSWScot A. Wolfe
- Jonathan K. WattsJWJonathan K. Watts
- Erik J. SontheimerESErik J. Sontheimer
Open Access
- 22 September 2026
- journal article
- Published by Elsevier in Cell Insight
- Vol. 5 (6)
Abstract
No abstract availableFunding Information
- Muscular Dystrophy Association
- Friedreich's Ataxia Research Alliance
- National Institutes of Health (R01CA275945)
- National Institutes of Health (R01GM150279)
- National Institutes of Health (S10OD036329)
- National Institutes of Health (P01HL158506)
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