Bioorthogonal Engineered Virus-Like Nanoparticles for Efficient Gene Therapy.

Bioorthogonal chemistry Gene therapy Recombinant biosome Site-specific codon mutation Virus-like nanoparticle

Journal

Nano-micro letters
ISSN: 2150-5551
Titre abrégé: Nanomicro Lett
Pays: Germany
ID NLM: 101727940

Informations de publication

Date de publication:
12 Aug 2023
Historique:
received: 10 04 2023
accepted: 20 06 2023
medline: 13 8 2023
pubmed: 13 8 2023
entrez: 12 8 2023
Statut: epublish

Résumé

Gene therapy offers potentially transformative strategies for major human diseases. However, one of the key challenges in gene therapy is developing an effective strategy that could deliver genes into the specific tissue. Here, we report a novel virus-like nanoparticle, the bioorthgonal engineered virus-like recombinant biosome (reBiosome), for efficient gene therapies of cancer and inflammatory diseases. The mutant virus-like biosome (mBiosome) is first prepared by site-specific codon mutation for displaying 4-azido-L-phenylalanine on vesicular stomatitis virus glycoprotein of eBiosome at a rational site, and the reBiosome is then prepared by clicking weak acid-responsive hydrophilic polymer onto the mBiosome via bioorthogonal chemistry. The results show that the reBiosome exhibits reduced virus-like immunogenicity, prolonged blood circulation time and enhanced gene delivery efficiency to weakly acidic foci (like tumor and arthritic tissue). Furthermore, reBiosome demonstrates robust therapeutic efficacy in breast cancer and arthritis by delivering gene editing and silencing systems, respectively. In conclusion, this study develops a universal, safe and efficient platform for gene therapies for cancer and inflammatory diseases.

Identifiants

pubmed: 37572220
doi: 10.1007/s40820-023-01153-y
pii: 10.1007/s40820-023-01153-y
pmc: PMC10423197
doi:

Types de publication

Journal Article

Langues

eng

Pagination

197

Informations de copyright

© 2023. Shanghai Jiao Tong University.

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Auteurs

Chun-Jie Bao (CJ)

State Key Laboratory of Natural and Biomimetic Drugs, Beijing Key Laboratory of Molecular Pharmaceutics and Drug Delivery Systems, and School of Pharmaceutical Sciences, Peking University, Beijing, 100191, People's Republic of China.
Jiangsu Collaborative Innovation Center of Chinese Medicinal Resources Industrialization, Nanjing University of Chinese Medicine, Nanjing, 210023, People's Republic of China.
School of Medicine & Holistic Integrative Medicine, Nanjing University of Chinese Medicine, Nanjing, 210023, People's Republic of China.

Jia-Lun Duan (JL)

State Key Laboratory of Natural and Biomimetic Drugs, Beijing Key Laboratory of Molecular Pharmaceutics and Drug Delivery Systems, and School of Pharmaceutical Sciences, Peking University, Beijing, 100191, People's Republic of China.
Jiangsu Collaborative Innovation Center of Chinese Medicinal Resources Industrialization, Nanjing University of Chinese Medicine, Nanjing, 210023, People's Republic of China.

Ying Xie (Y)

State Key Laboratory of Natural and Biomimetic Drugs, Beijing Key Laboratory of Molecular Pharmaceutics and Drug Delivery Systems, and School of Pharmaceutical Sciences, Peking University, Beijing, 100191, People's Republic of China.

Xin-Ping Feng (XP)

School of Chemical Sciences, University of Chinese Academy of Sciences, Beijing, 100049, People's Republic of China.

Wei Cui (W)

School of Chemical Sciences, University of Chinese Academy of Sciences, Beijing, 100049, People's Republic of China.

Song-Yue Chen (SY)

State Key Laboratory of Natural and Biomimetic Drugs, Beijing Key Laboratory of Molecular Pharmaceutics and Drug Delivery Systems, and School of Pharmaceutical Sciences, Peking University, Beijing, 100191, People's Republic of China.

Pei-Shan Li (PS)

State Key Laboratory of Natural and Biomimetic Drugs, Beijing Key Laboratory of Molecular Pharmaceutics and Drug Delivery Systems, and School of Pharmaceutical Sciences, Peking University, Beijing, 100191, People's Republic of China.

Yi-Xuan Liu (YX)

State Key Laboratory of Natural and Biomimetic Drugs, Beijing Key Laboratory of Molecular Pharmaceutics and Drug Delivery Systems, and School of Pharmaceutical Sciences, Peking University, Beijing, 100191, People's Republic of China.

Jin-Ling Wang (JL)

State Key Laboratory of Natural and Biomimetic Drugs, Beijing Key Laboratory of Molecular Pharmaceutics and Drug Delivery Systems, and School of Pharmaceutical Sciences, Peking University, Beijing, 100191, People's Republic of China.

Gui-Ling Wang (GL)

State Key Laboratory of Natural and Biomimetic Drugs, Beijing Key Laboratory of Molecular Pharmaceutics and Drug Delivery Systems, and School of Pharmaceutical Sciences, Peking University, Beijing, 100191, People's Republic of China.

Wan-Liang Lu (WL)

State Key Laboratory of Natural and Biomimetic Drugs, Beijing Key Laboratory of Molecular Pharmaceutics and Drug Delivery Systems, and School of Pharmaceutical Sciences, Peking University, Beijing, 100191, People's Republic of China. luwl@bjmu.edu.cn.

Classifications MeSH