About this Research Topic
Gene therapy involves the introduction of therapeutic genetic materials in order to rectify aberrant or mutated host gene products responsible for a disease. Delivery of the therapeutic gene requires the use of a vehicle, or 'vector'. The therapeutic gene is loaded onto the vector which then crosses the cell barrier to deliver the cargo gene to target cells, tissues or entire organs. The critical step in successful gene therapy is vector selection. Traditionally, modified viruses have been used as vectors, however non-viral vectors offer increased bio-safety, reduced pathogenicity, low cost and ease of production. Reduced delivery efficiency has left the potential of non-viral vectors relatively untapped.
This Research Topic will focus on materials as non-viral vectors for cellular uptake and delivery of genes and nucleic acids in order to achieve potential benefits to human health and advance gene therapy prospects. The aim of this Research Topic is to report current and future advancements in the use of materials, chemicals and particles as non-viral alternatives for nucleic acid delivery.
We welcome Original Research papers, Reviews, Mini-reviews or papers providing descriptions of novel methods for significantly advancing gene therapy. Themes of interest are limited to:
• Chemical gene/nucleic acid delivery systems (e.g. lipids, polymers, polyplexes, lipopolyplexes, silica, calcium phosphate)
• Engineered materials (e.g. MOFs, novel complexes, biobased/biomimetic materials, nanoparticles)
• Non-viral gene delivery as a research tool for investigating cellular processes
• Mechanisms of uptake and delivery
• Investigation of structure-function relationships between cargo nucleic acid and vector (e.g. genes, oligonucleotides, ncRNAs)
• Comparison with viral based systems
• Utility in various cell types, including expression in difficult to transfect cells (e.g. neurons or CAR-T cells)
This Research Topic will focus on materials as non-viral vectors for cellular uptake and delivery of genes and nucleic acids in order to achieve potential benefits to human health and advance gene therapy prospects. The aim of this Research Topic is to report current and future advancements in the use of materials, chemicals and particles as non-viral alternatives for nucleic acid delivery.
We welcome Original Research papers, Reviews, Mini-reviews or papers providing descriptions of novel methods for significantly advancing gene therapy. Themes of interest are limited to:
• Chemical gene/nucleic acid delivery systems (e.g. lipids, polymers, polyplexes, lipopolyplexes, silica, calcium phosphate)
• Engineered materials (e.g. MOFs, novel complexes, biobased/biomimetic materials, nanoparticles)
• Non-viral gene delivery as a research tool for investigating cellular processes
• Mechanisms of uptake and delivery
• Investigation of structure-function relationships between cargo nucleic acid and vector (e.g. genes, oligonucleotides, ncRNAs)
• Comparison with viral based systems
• Utility in various cell types, including expression in difficult to transfect cells (e.g. neurons or CAR-T cells)
Keywords: gene therapy, nucleic acid delivery, non-viral vectors, vectors
Important Note: All contributions to this Research Topic must be within the scope of the section and journal to which they are submitted, as defined in their mission statements. Frontiers reserves the right to guide an out-of-scope manuscript to a more suitable section or journal at any stage of peer review.
