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Ultrasonic particles: An approach for targeted gene delivery

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...

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Autores principales: Walsh, Aidan P.G., Gordon, Henry N., Peter, Karlheinz, Wang, Xiaowei
Formato: Online Artículo Texto
Lenguaje:English
Publicado: The Authors. Published by Elsevier B.V. 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8518240/
https://www.ncbi.nlm.nih.gov/pubmed/34662671
http://dx.doi.org/10.1016/j.addr.2021.113998
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author Walsh, Aidan P.G.
Gordon, Henry N.
Peter, Karlheinz
Wang, Xiaowei
author_facet Walsh, Aidan P.G.
Gordon, Henry N.
Peter, Karlheinz
Wang, Xiaowei
author_sort Walsh, Aidan P.G.
collection PubMed
description 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.
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spelling pubmed-85182402021-10-15 Ultrasonic particles: An approach for targeted gene delivery Walsh, Aidan P.G. Gordon, Henry N. Peter, Karlheinz Wang, Xiaowei Adv Drug Deliv Rev Article 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. The Authors. Published by Elsevier B.V. 2021-12 2021-10-15 /pmc/articles/PMC8518240/ /pubmed/34662671 http://dx.doi.org/10.1016/j.addr.2021.113998 Text en © 2021 The Authors Since January 2020 Elsevier has created a COVID-19 resource centre with free information in English and Mandarin on the novel coronavirus COVID-19. The COVID-19 resource centre is hosted on Elsevier Connect, the company's public news and information website. Elsevier hereby grants permission to make all its COVID-19-related research that is available on the COVID-19 resource centre - including this research content - immediately available in PubMed Central and other publicly funded repositories, such as the WHO COVID database with rights for unrestricted research re-use and analyses in any form or by any means with acknowledgement of the original source. These permissions are granted for free by Elsevier for as long as the COVID-19 resource centre remains active.
spellingShingle Article
Walsh, Aidan P.G.
Gordon, Henry N.
Peter, Karlheinz
Wang, Xiaowei
Ultrasonic particles: An approach for targeted gene delivery
title Ultrasonic particles: An approach for targeted gene delivery
title_full Ultrasonic particles: An approach for targeted gene delivery
title_fullStr Ultrasonic particles: An approach for targeted gene delivery
title_full_unstemmed Ultrasonic particles: An approach for targeted gene delivery
title_short Ultrasonic particles: An approach for targeted gene delivery
title_sort ultrasonic particles: an approach for targeted gene delivery
topic Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8518240/
https://www.ncbi.nlm.nih.gov/pubmed/34662671
http://dx.doi.org/10.1016/j.addr.2021.113998
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