Application of Gene Delivery: Challenges and Opportunities
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 July 2023) | Viewed by 3052
Special Issue Editors
Interests: gene therapy; non-viral vectors; drug delivery; 3D bioprinting; nanotechnology
Special Issues, Collections and Topics in MDPI journals
Interests: gene therapy; non-viral vectors; drug delivery; 3D bioprinting; nanotechnology
Special Issues, Collections and Topics in MDPI journals
Special Issue Information
Dear Colleagues,
Gene therapy has become one of the most promising strategies for the treatment of diseases with a genetic origin, or even acquired diseases, with many potential clinical applications.
Nucleic acid drugs are becoming essential therapeutic molecules for precision medicine using different approaches: overcominh the deficiency of a protein by the delivery of DNA vectors carrying the absent gene, the use of the antisense approach to block the translation of a specific mRNA or modulate RNA splicing, downregulating a gene of a selected mRNA by short interfering double-stranded RNAs (siRNAs), and the use of nucleic acids to block or interact with proteins.
More than 150 investigational new drug applications were filed for gene therapy in 2018 alone. Experts predict a growth in gene therapy approvals per year in the near future. This growth is set to come from ASOs and RNAi (Spinraza and Onpattro) and from potentially curative modalities deploying AAV and lentivirus therapies (Luxturna and Zynteglo). In addition, CRISPR gene-editing-based therapeutics present a long-term growth opportunity, generating significant excitement and investment in the technology. However, they are unlikely to have significant clinical impact in the short term, as further preclinical research is still needed.
Issues with delivery are still one of the most important problems to overcome in the development of nucleic acids as drugs. Although viral vectors such as retroviruses or adenoviruses have shown high transfection efficiencies and have been used in clinical trials, certain concerns regarding the immunogenicity or recombination of oncogenes must be overcome. Moreover, viral vectors still suffer from capacity constraints, a high cost of goods, long lead times, and significant upfront investment requirements. In contrast, nonviral vectors such as lipids, cell-penetrating peptides, polymers or gold nanoparticles have emerged as promising alternatives to deliver nucleic acids. It is essential to deepen the regulation of gene therapy drugs, as well as the investigation of the safety of these drugs.
Prof. Dr. Jon Zárate Sesma
Dr. Ilia Villate-Beitia
Guest Editors
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Keywords
- nanocarriers
- gene delivery
- nonviral vectors
- nucleic acids
- gene therapy
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