Ultrasonic particles: An approach for targeted gene delivery

Aidan P G Walsh; Henry N Gordon; Karlheinz Peter; Xiaowei Wang

doi:10.1016/j.addr.2021.113998

Ultrasonic particles: An approach for targeted gene delivery

Adv Drug Deliv Rev. 2021 Dec:179:113998. doi: 10.1016/j.addr.2021.113998. Epub 2021 Oct 15.

Authors

Aidan P G Walsh¹, Henry N Gordon², Karlheinz Peter³, Xiaowei Wang⁴

Affiliations

¹ Molecular Imaging and Theranostics Laboratory, Baker Heart and Diabetes Institute, Melbourne, VIC, Australia; Atherothrombosis and Vascular Biology Laboratory, Baker Heart and Diabetes Institute, Melbourne, VIC, Australia; Department of Medicine, Monash University, Melbourne, VIC, Australia.
² Molecular Imaging and Theranostics Laboratory, Baker Heart and Diabetes Institute, Melbourne, VIC, Australia; Atherothrombosis and Vascular Biology Laboratory, Baker Heart and Diabetes Institute, Melbourne, VIC, Australia; Department of Biochemistry and Pharmacology, University of Melbourne, VIC, Australia.
³ Atherothrombosis and Vascular Biology Laboratory, Baker Heart and Diabetes Institute, Melbourne, VIC, Australia; Department of Medicine, Monash University, Melbourne, VIC, Australia; Department of Cardiometabolic Health, University of Melbourne, VIC, Australia; La Trobe Institute for Molecular Science, La Trobe University, Melbourne, VIC, Australia.
⁴ Molecular Imaging and Theranostics Laboratory, Baker Heart and Diabetes Institute, Melbourne, VIC, Australia; Atherothrombosis and Vascular Biology Laboratory, Baker Heart and Diabetes Institute, Melbourne, VIC, Australia; Department of Medicine, Monash University, Melbourne, VIC, Australia; Department of Cardiometabolic Health, University of Melbourne, VIC, Australia; La Trobe Institute for Molecular Science, La Trobe University, Melbourne, VIC, Australia. Electronic address: xiaowei.wang@baker.edu.au.

Abstract

Gene therapy has been widely investigated for the treatment of genetic, acquired, and infectious diseases. Pioneering work utilized viral vectors; however, these are suspected of causing serious adverse events, resulting in the termination of several clinical trials. Non-viral vectors, such as lipid nanoparticles, have attracted significant interest, mainly due to their successful use in vaccines in the current COVID-19 pandemic. Although they allow safe delivery, they come with the disadvantage of off-target delivery. The application of ultrasound to ultrasound-sensitive particles allows for a direct, site-specific transfer of genetic materials into the organ/site of interest. This process, termed ultrasound-targeted gene delivery (UTGD), also increases cell membrane permeability and enhances gene uptake. This review focuses on the advances in ultrasound and the development of ultrasonic particles for UTGD across a range of diseases. Furthermore, we discuss the limitations and future perspectives of UTGD.

Keywords: Gene transfer; Microbubble; Nucleic acid; Sonoporation; Targeted therapy; Ultrasonic irradiation.

Publication types

Research Support, Non-U.S. Gov't
Review

MeSH terms

Animals
COVID-19
Gene Transfer Techniques*
Genetic Therapy / methods*
Humans
Liposomes
Molecular Targeted Therapy / methods*
Nanoparticles
Ultrasonics*

Substances

Lipid Nanoparticles
Liposomes