Emerging non-viral vectors for gene delivery
Abstract Gene therapy holds great promise for treating a multitude of inherited and acquired diseases by delivering functional genes, comprising DNA or RNA, into targeted cells or tissues to elicit manipulation of gene expression. However, the clinical implementation of gene therapy remains substant...
| Main Authors: | , , , , , , , , |
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| Format: | Article |
| Language: | English |
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BMC
2023-08-01
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| Series: | Journal of Nanobiotechnology |
| Subjects: | |
| Online Access: | https://doi.org/10.1186/s12951-023-02044-5 |
| _version_ | 1797451591872151552 |
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| author | Chenfei Wang Chaolan Pan Haiyang Yong Feifei Wang Tao Bo Yitong Zhao Bin Ma Wei He Ming Li |
| author_facet | Chenfei Wang Chaolan Pan Haiyang Yong Feifei Wang Tao Bo Yitong Zhao Bin Ma Wei He Ming Li |
| author_sort | Chenfei Wang |
| collection | DOAJ |
| description | Abstract Gene therapy holds great promise for treating a multitude of inherited and acquired diseases by delivering functional genes, comprising DNA or RNA, into targeted cells or tissues to elicit manipulation of gene expression. However, the clinical implementation of gene therapy remains substantially impeded by the lack of safe and efficient gene delivery vehicles. This review comprehensively outlines the novel fastest-growing and efficient non-viral gene delivery vectors, which include liposomes and lipid nanoparticles (LNPs), highly branched poly(β-amino ester) (HPAE), single-chain cyclic polymer (SCKP), poly(amidoamine) (PAMAM) dendrimers, and polyethyleneimine (PEI). Particularly, we discuss the research progress, potential development directions, and remaining challenges. Additionally, we provide a comprehensive overview of the currently approved non-viral gene therapeutics, as well as ongoing clinical trials. With advances in biomedicine, molecular biology, materials science, non-viral gene vectors play an ever-expanding and noteworthy role in clinical gene therapy. |
| first_indexed | 2024-03-09T14:56:52Z |
| format | Article |
| id | doaj.art-252ecbf823b04950a8f87266a0ad02a9 |
| institution | Directory Open Access Journal |
| issn | 1477-3155 |
| language | English |
| last_indexed | 2024-03-09T14:56:52Z |
| publishDate | 2023-08-01 |
| publisher | BMC |
| record_format | Article |
| series | Journal of Nanobiotechnology |
| spelling | doaj.art-252ecbf823b04950a8f87266a0ad02a92023-11-26T14:08:09ZengBMCJournal of Nanobiotechnology1477-31552023-08-0121111810.1186/s12951-023-02044-5Emerging non-viral vectors for gene deliveryChenfei Wang0Chaolan Pan1Haiyang Yong2Feifei Wang3Tao Bo4Yitong Zhao5Bin Ma6Wei He7Ming Li8Department of Dermatology, Children’s Hospital of Fudan University, National Children’s Medical CenterDepartment of Dermatology, Children’s Hospital of Fudan University, National Children’s Medical CenterSchool of Chemical Engineering and Technology, Xi’an Jiaotong UniversityDepartment of Anesthesiology, The Second Affiliated Hospital of Air Force Medical UniversitySchool of Basic Medical Sciences, Fudan UniversitySchool of Medicine, Anhui University of Science and TechnologySchool of Chemical Engineering and Technology, Xi’an Jiaotong UniversitySchool of Medicine, Anhui University of Science and TechnologyDepartment of Dermatology, Children’s Hospital of Fudan University, National Children’s Medical CenterAbstract Gene therapy holds great promise for treating a multitude of inherited and acquired diseases by delivering functional genes, comprising DNA or RNA, into targeted cells or tissues to elicit manipulation of gene expression. However, the clinical implementation of gene therapy remains substantially impeded by the lack of safe and efficient gene delivery vehicles. This review comprehensively outlines the novel fastest-growing and efficient non-viral gene delivery vectors, which include liposomes and lipid nanoparticles (LNPs), highly branched poly(β-amino ester) (HPAE), single-chain cyclic polymer (SCKP), poly(amidoamine) (PAMAM) dendrimers, and polyethyleneimine (PEI). Particularly, we discuss the research progress, potential development directions, and remaining challenges. Additionally, we provide a comprehensive overview of the currently approved non-viral gene therapeutics, as well as ongoing clinical trials. With advances in biomedicine, molecular biology, materials science, non-viral gene vectors play an ever-expanding and noteworthy role in clinical gene therapy.https://doi.org/10.1186/s12951-023-02044-5Gene therapyGene delivery processNon-viral gene vectorsNanomaterialsLipid nanoparticles |
| spellingShingle | Chenfei Wang Chaolan Pan Haiyang Yong Feifei Wang Tao Bo Yitong Zhao Bin Ma Wei He Ming Li Emerging non-viral vectors for gene delivery Journal of Nanobiotechnology Gene therapy Gene delivery process Non-viral gene vectors Nanomaterials Lipid nanoparticles |
| title | Emerging non-viral vectors for gene delivery |
| title_full | Emerging non-viral vectors for gene delivery |
| title_fullStr | Emerging non-viral vectors for gene delivery |
| title_full_unstemmed | Emerging non-viral vectors for gene delivery |
| title_short | Emerging non-viral vectors for gene delivery |
| title_sort | emerging non viral vectors for gene delivery |
| topic | Gene therapy Gene delivery process Non-viral gene vectors Nanomaterials Lipid nanoparticles |
| url | https://doi.org/10.1186/s12951-023-02044-5 |
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