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J Control Release. 2021 Dec 02; doi: 10.1016/j.jconrel.2021.11.046. Epub 2021 Dec 02.

Enhanced nuclear gene delivery via integrating and streamlining intracellular pathway.

Journal of controlled release : official journal of the Controlled Release Society

Lian-Yu Qi, Yi Wang, Li-Fan Hu, Pu-Song Zhao, Hao-Yuan Yu, Lei Xing, Xiang-Dong Gao, Qing-Ri Cao, Hu-Lin Jiang

Affiliations

  1. State Key Laboratory of Natural Medicines, Department of Pharmaceutics, China Pharmaceutical University, Nanjing 210009, China.
  2. State Key Laboratory of Natural Medicines, Department of Pharmaceutics, China Pharmaceutical University, Nanjing 210009, China; Jiangsu Key Laboratory of Druggability of Biopharmaceuticals, China Pharmaceutical University, Nanjing 210009, China; Jiangsu Key Laboratory of Drug Screening, China Pharmaceutical University, Nanjing 210009, China; NMPA Key Laboratory for Research and Evaluation of Pharmaceutical Preparations and Excipients, China Pharmaceutical University, Nanjing 210009, China.
  3. State Key Laboratory of Natural Medicines, Department of Pharmaceutics, China Pharmaceutical University, Nanjing 210009, China; Jiangsu Key Laboratory of Druggability of Biopharmaceuticals, China Pharmaceutical University, Nanjing 210009, China. Electronic address: [email protected].
  4. College of Pharmaceutical Sciences, Soochow University, Suzhou 215123, China. Electronic address: [email protected].
  5. State Key Laboratory of Natural Medicines, Department of Pharmaceutics, China Pharmaceutical University, Nanjing 210009, China; Jiangsu Key Laboratory of Druggability of Biopharmaceuticals, China Pharmaceutical University, Nanjing 210009, China; Jiangsu Key Laboratory of Drug Screening, China Pharmaceutical University, Nanjing 210009, China; NMPA Key Laboratory for Research and Evaluation of Pharmaceutical Preparations and Excipients, China Pharmaceutical University, Nanjing 210009, China. Electronic address: [email protected].

PMID: 34864117 DOI: 10.1016/j.jconrel.2021.11.046

Abstract

The essential challenge of gene therapy is to develop safe and efficient vectors that escort genes to target sites. However, due to the cumbersome workflow of gene transfection into cells, successive gene loss occurs. This leads to considerable reductions in nuclear gene uptake, eventually causing low gene expression. Herein, we designed a gene vector named CA

Copyright © 2021. Published by Elsevier B.V.

Keywords: Caveolae-mediated pathway; Endoplasmic reticulum-targeting; Gene therapy; Lysosomal escape; Nuclear delivery

Conflict of interest statement

Declaration of Competing Interest The authors declare no competing financial interest.

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