Editorial Board Members’ Collection Series: Progress and Challenges of Gene Delivery Systems

A special issue of Pharmaceutics (ISSN 1999-4923). This special issue belongs to the section "Gene and Cell Therapy".

Deadline for manuscript submissions: closed (10 April 2024) | Viewed by 2492

Special Issue Editors

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Guest Editor
Department of Pharmaceutics, School of Pharmaceutical Sciences, Peking University, Beijing 100191, China
Interests: targeting drug delivery; nanotechnology and biotechnology

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Guest Editor
College of Pharmacy and Nutrition, University of Saskatchewan, 107 Wiggins Road, Health Sciences Building, Room 3D01.5, Box 3D01-13, Saskatoon, SK S7N 5E5, Canada
Interests: nucleic acid delivery; nanodiamonds; cationic gemini lipids; self-assembling nanoparticles; small angle X-ray scattering; flow cytometry; radiopharmaceuticals
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Special Issue Information

Dear Colleagues,

In the last few decades, nucleic acid-based therapy has achieved rapid development leading to the approval of some gene therapeutics. However, the instability, short half-life, and membrane impermeability severely hinder the efficient delivery of gene drugs. Development of safe and efficient delivery systems remains the key issue to advance gene therapy. Viral vectors and non-viral vectors, the two main delivery systems at present, have their own advantages and disadvantages. Nanomaterial-based non-viral vectors, including polymers, dendrimers, liposomes, lipid nanoparticles, peptides, and inorganic nanoparticles, have the advantages of safety, non-immunogenicity, and ease of massive production and have been extensively explored for gene therapy. To promote their clinical translation, researchers have tried various strategies to improve delivery efficiency and reduce off-target effects. This issue focuses on the latest progress of nucleic acid delivery systems and highlights the challenges for their translation into clinic, aiming to present a fundamental understanding of this field.

Prof. Dr. Xianrong Qi
Prof. Dr. Ildiko Badea
Guest Editors

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26 pages, 1863 KiB  
Advances and Challenges of Stimuli-Responsive Nucleic Acids Delivery System in Gene Therapy
by Meng Lin and Xianrong Qi
Pharmaceutics 2023, 15(5), 1450; https://doi.org/10.3390/pharmaceutics15051450 - 10 May 2023
Cited by 5 | Viewed by 2064
Gene therapy has emerged as a powerful tool to treat various diseases, such as cardiovascular diseases, neurological diseases, ocular diseases and cancer diseases. In 2018, the FDA approved Patisiran (the siRNA therapeutic) for treating amyloidosis. Compared with traditional drugs, gene therapy can directly [...] Read more.
Gene therapy has emerged as a powerful tool to treat various diseases, such as cardiovascular diseases, neurological diseases, ocular diseases and cancer diseases. In 2018, the FDA approved Patisiran (the siRNA therapeutic) for treating amyloidosis. Compared with traditional drugs, gene therapy can directly correct the disease-related genes at the genetic level, which guarantees a sustained effect. However, nucleic acids are unstable in circulation and have short half-lives. They cannot pass through biological membranes due to their high molecular weight and massive negative charges. To facilitate the delivery of nucleic acids, it is crucial to develop a suitable delivery strategy. The rapid development of delivery systems has brought light to the gene delivery field, which can overcome multiple extracellular and intracellular barriers that prevent the efficient delivery of nucleic acids. Moreover, the emergence of stimuli-responsive delivery systems has made it possible to control the release of nucleic acids in an intelligent manner and to precisely guide the therapeutic nucleic acids to the target site. Considering the unique properties of stimuli-responsive delivery systems, various stimuli-responsive nanocarriers have been developed. For example, taking advantage of the physiological variations of a tumor (pH, redox and enzymes), various biostimuli- or endogenous stimuli-responsive delivery systems have been fabricated to control the gene delivery processes in an intelligent manner. In addition, other external stimuli, such as light, magnetic fields and ultrasound, have also been employed to construct stimuli-responsive nanocarriers. Nevertheless, most stimuli-responsive delivery systems are in the preclinical stage, and some critical issues remain to be solved for advancing the clinical translation of these nanocarriers, such as the unsatisfactory transfection efficiency, safety issues, complexity of manufacturing and off-target effects. The purpose of this review is to elaborate the principles of stimuli-responsive nanocarriers and to emphasize the most influential advances of stimuli-responsive gene delivery systems. Current challenges of their clinical translation and corresponding solutions will also be highlighted, which will accelerate the translation of stimuli-responsive nanocarriers and advance the development of gene therapy. Full article
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