Advances in Targeted Gene Delivery

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

Deadline for manuscript submissions: closed (20 September 2022) | Viewed by 4666

Special Issue Editor


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Guest Editor
National Engineering Research Center for Biomaterials, Sichuan University, Chengdu 610064, China
Interests: gene therapy, targeted gene delivery, biomaterial

Special Issue Information

Dear Colleagues,

Efficiently delivering functional genes to target sites is the determinant factor for successful applications, especially for in vivo gene therapy, genome editing, cell reprogramming, and vaccination. Meanwhile, it has also been one of the major obstacles to in vivo applications for years. Recent advances in targeted gene delivery have acquired significant progress in the fields of pharmaceutics, biomaterials, biology, medicine, and bioengineering. This Special Issue of Pharmaceutics will address the latest advances in targeted gene delivery, focusing on innovative strategies, advanced technologies, smart gene carrier materials, new medical devices, and therapeutic trials/applications. We kindly invite you to submit a manuscript for this Special Issue. Full papers, communications, and reviews are all welcome.

Submissions can cover the following topics (but are not limited to them):

  • Technologies for targeted delivery of DNA/RNA (mRNA/siRNA/miRNA/ncRNA, etc.)/nucleic acid vaccines;
  • Functionalized materials for targeted gene delivery;
  • Gene delivery to target tissues/organs;
  • Gene delivery to target cells;
  • Targeted gene delivery to organelles/molecules/genome;
  • Therapeutic trials/cases with targeted gene delivery;
  • Vaccination via targeted gene delivery.

Prof. Dr. Gang Wang
Guest Editor

Manuscript Submission Information

Manuscripts should be submitted online at www.mdpi.com by registering and logging in to this website. Once you are registered, click here to go to the submission form. Manuscripts can be submitted until the deadline. All submissions that pass pre-check are peer-reviewed. Accepted papers will be published continuously in the journal (as soon as accepted) and will be listed together on the special issue website. Research articles, review articles as well as short communications are invited. For planned papers, a title and short abstract (about 100 words) can be sent to the Editorial Office for announcement on this website.

Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). All manuscripts are thoroughly refereed through a single-blind peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. Pharmaceutics is an international peer-reviewed open access monthly journal published by MDPI.

Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 2900 CHF (Swiss Francs). Submitted papers should be well formatted and use good English. Authors may use MDPI's English editing service prior to publication or during author revisions.

Keywords

  • targeted gene delivery
  • gene therapy
  • biomaterial
  • gene carrier/vector
  • functional gene
  • mRNA/siRNA/miRNA
  • vaccination

Published Papers (2 papers)

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Research

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16 pages, 3011 KiB  
Article
Development of a Combined Lipid-Based Nanoparticle Formulation for Enhanced siRNA Delivery to Vascular Endothelial Cells
by Yutong He, Dongdong Bi, Josée A. Plantinga, Grietje Molema, Jeroen Bussmann and Jan A. A. M. Kamps
Pharmaceutics 2022, 14(10), 2086; https://doi.org/10.3390/pharmaceutics14102086 - 29 Sep 2022
Cited by 8 | Viewed by 2652
Abstract
Low transfection efficiency in endothelial cells (EC) is still a bottleneck for the majority of siRNA-based vascular delivery approaches. In this work, we developed a lipid-based nanoparticle (LNP) formulation based on a combination of a permanently charged cationic lipid-DOTAP and a conditionally ionized [...] Read more.
Low transfection efficiency in endothelial cells (EC) is still a bottleneck for the majority of siRNA-based vascular delivery approaches. In this work, we developed a lipid-based nanoparticle (LNP) formulation based on a combination of a permanently charged cationic lipid-DOTAP and a conditionally ionized cationic lipid-MC3 (DOTAP/MC3) for the enhanced delivery of siRNA into EC. Compared with a single DOTAP or MC3-based benchmark LNP, we demonstrated that the DOTAP/MC3 LNP formulation shows the best transfection efficiency both in primary EC in vitro and in endothelium in zebrafish. The high transfection activity of the DOTAP/MC3 LNP formulation is achieved by a combination of improved endothelial association mediated by DOTAP and MC3-triggered efficient siRNA intracellular release in EC. Furthermore, AbVCAM-1-coupled DOTAP/MC3 LNP-mediated siRNARelA transfection showed pronounced anti-inflammatory effects in inflammatory-activated primary EC by effectively blocking the NF-κB pathway. In conclusion, the combination of permanent and ionizable cationic lipids in LNP formulation provides an effective endothelial cell delivery of siRNA. Full article
(This article belongs to the Special Issue Advances in Targeted Gene Delivery)
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Review

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23 pages, 3071 KiB  
Review
The Progress of Non-Viral Materials and Methods for Gene Delivery to Skeletal Muscle
by Zhanpeng Cui, Yang Jiao, Linyu Pu, James Zhenggui Tang and Gang Wang
Pharmaceutics 2022, 14(11), 2428; https://doi.org/10.3390/pharmaceutics14112428 - 10 Nov 2022
Cited by 3 | Viewed by 1646
Abstract
Since Jon A. Wolff found skeletal muscle cells being able to express foreign genes and Russell J. Mumper increased the gene transfection efficiency into the myocytes by adding polymers, skeletal muscles have become a potential gene delivery and expression target. Different methods have [...] Read more.
Since Jon A. Wolff found skeletal muscle cells being able to express foreign genes and Russell J. Mumper increased the gene transfection efficiency into the myocytes by adding polymers, skeletal muscles have become a potential gene delivery and expression target. Different methods have been developing to deliver transgene into skeletal muscles. Among them, viral vectors may achieve potent gene delivery efficiency. However, the potential for triggering biosafety risks limited their clinical applications. Therefore, non-viral biomaterial-mediated methods with reliable biocompatibility are promising tools for intramuscular gene delivery in situ. In recent years, a series of advanced non-viral gene delivery materials and related methods have been reported, such as polymers, liposomes, cell penetrating peptides, as well as physical delivery methods. In this review, we summarized the research progresses and challenges in non-viral intramuscular gene delivery materials and related methods, focusing on the achievements and future directions of polymers. Full article
(This article belongs to the Special Issue Advances in Targeted Gene Delivery)
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