Targeted Delivery of Nucleic Acids
A special issue of International Journal of Molecular Sciences (ISSN 1422-0067). This special issue belongs to the section "Molecular Biology".
Deadline for manuscript submissions: 28 August 2024 | Viewed by 374
Special Issue Editor
Special Issue Information
Dear Colleagues,
In modern medicine, the delivery of nucleic acids such as pDNA, siRNA, miRNA, mRNA and the CRISPR-Cas system is a crucial process for gene therapy, vaccine development and cancer treatment. These materials can regulate or modify genetic information within cells, potentially treating diseases at their root. mRNA, for instance, has shown significant preventative effects against infectious diseases such as COVID-19 and is being investigated for non-infectious diseases such as cancer. The CRISPR-Cas system, known for gene editing, has gained FDA approval, representing a breakthrough in treating genetic disorders.
However, delivering these nucleic acids presents some challenges. They are unstable outside cells, can trigger immune responses or degrade quickly in the body. Ensuring effective delivery to specific cells or tissues is difficult. The off-targeting of genetic materials may demand over-dosing to meet therapeutic effectiveness, which usually results in aberrant side effects.
Targeted delivery systems are a promising solution, ensuring that nucleic acids are delivered directly to specific cells or tissues, thus maximizing treatment efficacy and minimizing side effects. For example, nanoparticles targeting cancer cell-specific receptors can exclusively deliver nucleic acids to cancer cells, essential for precision treatments like gene editing.
The targeted delivery of nucleic acids is significant in clinical research and treatment. Many nucleic acid-based therapeutics are in clinical trials, with some approved for use. mRNA vaccines have effectively triggered immune responses against specific viruses, and gene therapies using CRISPR-Cas have shown potential in treating genetic disorders. The evolution of these technologies is expected to enhance the precision and safety of targeted delivery systems further.
Dr. Yong-Serk Park
Guest Editor
Manuscript Submission Information
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Keywords
- nucleic acid delivery
- gene delivery
- pDNA delivery
- siRNA delivery
- miRNA delivery
- mRNA delivery
- CRISPR-Cas delivery
- nanoparticle
- lipid nanoparticle
- targeted therapy
