Gene therapy for monogenic disorders: challenges, strategies, and perspectives

Yi Zhang; Zhi-Ying Wu

doi:10.1016/j.jgg.2023.08.001

Gene therapy for monogenic disorders: challenges, strategies, and perspectives

doi: 10.1016/j.jgg.2023.08.001

Yi Zhang,
Zhi-Ying Wu^,

Department of Medical Genetics and Center for Rare Diseases, and Department of Neurology in Second Affiliated Hospital, and Key Laboratory of Medical Neurobiology of Zhejiang Province, Zhejiang University School of Medicine, Hangzhou, Zhejiang 310009, China

详细信息

通讯作者:
Zhi-Ying Wu,Email address:zhiyingwu@zju.edu.cn

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- 文章访问数: 130
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- 被引次数: 0
出版历程
- 收稿日期: 2023-06-03
- 录用日期: 2023-08-14
- 修回日期: 2023-08-02
- 网络出版日期: 2023-08-15

Gene therapy for monogenic disorders: challenges, strategies, and perspectives

Yi Zhang,
Zhi-Ying Wu^,

Department of Medical Genetics and Center for Rare Diseases, and Department of Neurology in Second Affiliated Hospital, and Key Laboratory of Medical Neurobiology of Zhejiang Province, Zhejiang University School of Medicine, Hangzhou, Zhejiang 310009, China

摘要

摘要:
Monogenic disorders refer to a group of human diseases caused by mutations in single genes. While disease-modifying therapies have offered some relief to symptoms and delayed progression for some monogenic diseases, most of these diseases still lack effective treatments. In recent decades, gene therapy has emerged as a promising therapeutic strategy for genetic disorders. Researchers have developed various gene manipulation tools and gene delivery systems to treat monogenic diseases. Despite this progress, concerns about inefficient delivery, persistent expression, immunogenicity, toxicity, capacity limitation, genomic integration, and limited tissue specificity still need to be addressed. This review gives an overview of commonly used gene therapy and delivery tools, along with the challenges they face and the potential strategies to counter them.
- Gene therapy /
- Monogenic diseases /
- Gene editing /
- Gene delivery /
- Challenges
Abstract:
Monogenic disorders refer to a group of human diseases caused by mutations in single genes. While disease-modifying therapies have offered some relief to symptoms and delayed progression for some monogenic diseases, most of these diseases still lack effective treatments. In recent decades, gene therapy has emerged as a promising therapeutic strategy for genetic disorders. Researchers have developed various gene manipulation tools and gene delivery systems to treat monogenic diseases. Despite this progress, concerns about inefficient delivery, persistent expression, immunogenicity, toxicity, capacity limitation, genomic integration, and limited tissue specificity still need to be addressed. This review gives an overview of commonly used gene therapy and delivery tools, along with the challenges they face and the potential strategies to counter them.
- Gene therapy /
- Monogenic diseases /
- Gene editing /
- Gene delivery /
- Challenges

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