TY - JOUR
T1 - Nanotechnology-enabled gene delivery for cancer and other genetic diseases
AU - Jiang, Tong
AU - Gonzalez, Karina Marie
AU - Cordova, Leyla Estrella
AU - Lu, Jianqin
N1 - Funding Information: This work was supported in part by a Startup Fund from the R. Ken Coit College of Pharmacy at The University of Arizona and a PhRMA Foundation Research Starter Grant in Drug Delivery, and by National Institutes of Health (NIH) grants (R35GM147002 and R01CA272487). Publisher Copyright: © 2023 Informa UK Limited, trading as Taylor & Francis Group.
PY - 2023
Y1 - 2023
N2 - Introduction: Despite gene therapy is ideal for genetic abnormality-related diseases, the easy degradation, poor targeting, and inefficiency in entering targeted cells are plaguing the effective delivery of gene therapy. Viral and non-viral vectors have been used for delivering gene therapeutics in vivo by safeguarding nucleic acid agents to target cells and to reach the specific intracellular location. A variety of nanotechnology-enabled safe and efficient systems have been successfully developed to improve the targeting ability for effective therapeutic delivery of genetic drugs. Areas covered: In this review, we outline the multiple biological barriers associated with gene delivery process, and highlight recent advances to gene therapy strategy in vivo, including gene correction, gene silencing, gene activation and genome editing. We point out current developments and challenges exist of non-viral and viral vector systems in association with chemical and physical gene delivery technologies and their potential for the future. Expert opinion: This review focuses on the opportunities and challenges to various gene therapy strategy, with specific emphasis on overcoming the challenges through the development of biocompatibility and smart gene vectors for potential clinical application.
AB - Introduction: Despite gene therapy is ideal for genetic abnormality-related diseases, the easy degradation, poor targeting, and inefficiency in entering targeted cells are plaguing the effective delivery of gene therapy. Viral and non-viral vectors have been used for delivering gene therapeutics in vivo by safeguarding nucleic acid agents to target cells and to reach the specific intracellular location. A variety of nanotechnology-enabled safe and efficient systems have been successfully developed to improve the targeting ability for effective therapeutic delivery of genetic drugs. Areas covered: In this review, we outline the multiple biological barriers associated with gene delivery process, and highlight recent advances to gene therapy strategy in vivo, including gene correction, gene silencing, gene activation and genome editing. We point out current developments and challenges exist of non-viral and viral vector systems in association with chemical and physical gene delivery technologies and their potential for the future. Expert opinion: This review focuses on the opportunities and challenges to various gene therapy strategy, with specific emphasis on overcoming the challenges through the development of biocompatibility and smart gene vectors for potential clinical application.
KW - Gene therapy
KW - drug delivery system
KW - nanotechnology
KW - non-viral vectors
KW - viral vectors
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U2 - 10.1080/17425247.2023.2200246
DO - 10.1080/17425247.2023.2200246
M3 - Review article
C2 - 37017558
SN - 1742-5247
VL - 20
SP - 523
EP - 540
JO - Expert Opinion on Drug Delivery
JF - Expert Opinion on Drug Delivery
IS - 4
ER -