Targeted Delivery of Genes
A special issue of Pharmaceutics (ISSN 1999-4923). This special issue belongs to the section "Gene and Cell Therapy".
Deadline for manuscript submissions: 20 January 2025 | Viewed by 98
Special Issue Editors
Interests: tissue engineering; gene therapy; orthopaedics; cancer; mesenchymal stem cells
Special Issue Information
Dear Colleagues,
Achieving target-specific delivery of oligonucleotides, whether to disease sites, specific tissues, or organs, is crucial for enhancing therapeutic precision and minimizing off-target effects. This precise delivery, facilitated by advanced methods like chemically modified oligonucleotides and nanoparticles, holds immense promise for improving treatment efficacy. Oligonucleotides and genes, particularly mRNA, siRNA, antisense RNA, and the CRISPR-Cas9 system, hold promise as alternatives to conventional therapeutic modalities. In 2023, we witnessed approvals of seven new cell and gene therapies (five of these for the treatment of rare genetic diseases). However, these genetic materials are inherently unstable in the body and require a sophisticated delivery system for effective results. The choice of delivery tools for oligonucleotides, including peptides, lipid nanoparticles (LNPs), and proteins, is paramount in ensuring their therapeutic effectiveness. These tools serve as carriers, guiding oligonucleotides to their intended destinations within the body, such as specific tissues or cells. Peptides and proteins can enhance cellular uptake, while LNPs provide stability and protection for oligonucleotides. Selecting the appropriate delivery tool is crucial for achieving targeted and efficient delivery, maximizing the potential of oligonucleotide-based therapies, and minimizing adverse effects.
This Special Issue aims to compile research articles and reviews spotlighting cancer-targeted, tissue-targeted, and organ-targeted delivery of oligonucleotides. Special emphasis is placed on modified mRNA, siRNA, antisense RNA, CRISPR-Cas9, microRNA, plasmid DNA and DNA, along with their effective delivery systems involving nanoparticles, dendrimers, and LNPs. Understanding the structural and chemical modification of oligonucleotides, coupled with successful packaging systems, is paramount for the success of gene therapy in this rapidly advancing field.
Dr. Caroline Curtin
Dr. Anjuman Ara Begum
Guest Editors
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Keywords
- targeting
- disease targeting
- organ targeting
- tissue or cell targeting
- oligonucleotides
- mRNA
- CRISPR
- nanoparticle
- lipid nanoparticle (LNP)
- delivery system
- siRNA
- microRNA
- antisense RNA
- plasmid DNA
- delivery vector
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