Ultrasound-targeted microbubble destruction in gene therapy: A new tool to cure human diseases

Human gene therapy has made significant advances in less than two decades. Within this short period of time, gene therapy has proceeded from the conceptual stage to technology development and laboratory research, and finally to clinical trials for the treatment of a variety of deadly diseases. Cardi...

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Main Authors: Jun Wu, Ren-Ke Li
Format: Article
Language:English
Published: KeAi Communications Co., Ltd. 2017-06-01
Series:Genes and Diseases
Subjects:
Online Access:http://www.sciencedirect.com/science/article/pii/S2352304216300344
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author Jun Wu
Ren-Ke Li
author_facet Jun Wu
Ren-Ke Li
author_sort Jun Wu
collection DOAJ
description Human gene therapy has made significant advances in less than two decades. Within this short period of time, gene therapy has proceeded from the conceptual stage to technology development and laboratory research, and finally to clinical trials for the treatment of a variety of deadly diseases. Cardiovascular disease, cancer, and stroke are leading causes of death worldwide. Despite advances in medical, interventional, radiation and surgical treatments, the mortality rate remains high, and the need for novel therapies is great. Gene therapy provides an efficient approach to disease treatment. Notable advances in gene therapy have been made for genetic disorders, including severe combined immune deficiency, chronic granulomatus disorder, hemophilia and blindness, as well as for acquired diseases, including cancer and neurodegenerative and cardiovascular diseases. However, lack of an efficient delivery system to target cells as well as the difficulty of sustained expression of transgenes has hindered advancements in gene therapy. Ultrasound targeted microbubble destruction (UTMD) is a promising approach for target-specific gene delivery, and it has been successfully investigated for the treatment of many diseases in the past decade. In this paper, we review UTMD-mediated gene delivery for the treatment of cardiovascular diseases, cancer and stroke.
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spelling doaj.art-c9770c833be24274b0a9c25aec77cdbd2023-09-03T02:01:04ZengKeAi Communications Co., Ltd.Genes and Diseases2352-30422017-06-0142647410.1016/j.gendis.2016.08.001Ultrasound-targeted microbubble destruction in gene therapy: A new tool to cure human diseasesJun Wu0Ren-Ke Li1Toronto General Research Institute, Division of Cardiovascular Surgery, University Health Network, Toronto, Ontario, CanadaToronto General Research Institute, Division of Cardiovascular Surgery, University Health Network, Toronto, Ontario, CanadaHuman gene therapy has made significant advances in less than two decades. Within this short period of time, gene therapy has proceeded from the conceptual stage to technology development and laboratory research, and finally to clinical trials for the treatment of a variety of deadly diseases. Cardiovascular disease, cancer, and stroke are leading causes of death worldwide. Despite advances in medical, interventional, radiation and surgical treatments, the mortality rate remains high, and the need for novel therapies is great. Gene therapy provides an efficient approach to disease treatment. Notable advances in gene therapy have been made for genetic disorders, including severe combined immune deficiency, chronic granulomatus disorder, hemophilia and blindness, as well as for acquired diseases, including cancer and neurodegenerative and cardiovascular diseases. However, lack of an efficient delivery system to target cells as well as the difficulty of sustained expression of transgenes has hindered advancements in gene therapy. Ultrasound targeted microbubble destruction (UTMD) is a promising approach for target-specific gene delivery, and it has been successfully investigated for the treatment of many diseases in the past decade. In this paper, we review UTMD-mediated gene delivery for the treatment of cardiovascular diseases, cancer and stroke.http://www.sciencedirect.com/science/article/pii/S2352304216300344Gene therapyCardiovascular diseasesCancerStrokeUTMD
spellingShingle Jun Wu
Ren-Ke Li
Ultrasound-targeted microbubble destruction in gene therapy: A new tool to cure human diseases
Genes and Diseases
Gene therapy
Cardiovascular diseases
Cancer
Stroke
UTMD
title Ultrasound-targeted microbubble destruction in gene therapy: A new tool to cure human diseases
title_full Ultrasound-targeted microbubble destruction in gene therapy: A new tool to cure human diseases
title_fullStr Ultrasound-targeted microbubble destruction in gene therapy: A new tool to cure human diseases
title_full_unstemmed Ultrasound-targeted microbubble destruction in gene therapy: A new tool to cure human diseases
title_short Ultrasound-targeted microbubble destruction in gene therapy: A new tool to cure human diseases
title_sort ultrasound targeted microbubble destruction in gene therapy a new tool to cure human diseases
topic Gene therapy
Cardiovascular diseases
Cancer
Stroke
UTMD
url http://www.sciencedirect.com/science/article/pii/S2352304216300344
work_keys_str_mv AT junwu ultrasoundtargetedmicrobubbledestructioningenetherapyanewtooltocurehumandiseases
AT renkeli ultrasoundtargetedmicrobubbledestructioningenetherapyanewtooltocurehumandiseases