Focused ultrasound-mediated lipid nanoparticle delivery for brain gene editing

Molecular Therapy — Nucleic Acids 2026 Open access

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Authors: Kaiyuan Zheng, Fotios N. Tsitsos, Elisa E. Konofagou, Kam W. Leong
DOI: 10.1016/j.omtn.2026.103012
Cited by: 1
References: 29

Abstract

mRNA and apoE4 protein expression, and attenuated astrocytes and microglial activation. These results establish FUS-mediated pDNA-LNP delivery as a non-invasive, non-viral strategy for brain gene editing that provides spatial control and cell-type-specific expression, while accommodating large genetic payload and enabling repeatable dosing.

Fields of Study & Subjects

Ultrasound and Hyperthermia Applications Microbial Inactivation Methods Ultrasound and Cavitation Phenomena

Publication details

Journal / source
Molecular Therapy — Nucleic Acids
Publisher
Elsevier BV
Volume
37
Issue
3
Pages
103012–103012
Publication date
14 July 2026
Language
EN