Gene Delivery
Gene Delivery refers to the use of affinity-functionalized nanotechnology platforms tailored for the precise delivery of genetic material—such as plasmid DNA, mRNA, siRNA, or CRISPR components—to specific cells or tissues.
Technical Content and Mechanism
- Nanocarrier Design for Gene Delivery:
- Material Selection:
Nanocarriers for gene delivery are often composed of biodegradable polymers (e.g., poly(lactic-co-glycolic acid) [PLGA], chitosan, polyethyleneimine [PEI]), lipid-based systems (liposomes, lipid nanoparticles), or inorganic nanoparticles (e.g., gold, silica).
- Material Selection:
- Affinity Functionalization:
- Optimizing Ligand Display:
The density, orientation, and spatial distribution of ligands are optimized to maximize binding to target cells while preventing steric hindrance, ensuring efficient receptor-mediated recognition and uptake.
- Optimizing Ligand Display:
- Genetic Payload Loading and Protection:
- Complexation or Encapsulation:
Genetic material may be electrostatically complexed with cationic polymers or encapsulated within lipid or polymeric matrices. This protects nucleic acids from nuclease degradation and immune recognition during circulation.
- Complexation or Encapsulation:
Applications
- Gene Therapy:
Targeted delivery of therapeutic genes can correct genetic defects, treat cancers, or modulate disease-related pathways by introducing, silencing, or editing specific genes in affected tissues with high precision. - RNA-Based Therapies:
Delivery of mRNA for protein replacement, siRNA for gene silencing, or CRISPR components for genome editing to specific cells can treat a variety of diseases, including genetic disorders, cancers, and viral infections, while minimizing off-target effects. - Vaccination and Immunotherapy:
Delivery of genetic material encoding antigens directly into dendritic cells or other antigen-presenting cells can generate strong and specific immune responses, forming the basis of DNA/RNA vaccines and personalized immunotherapies.
Gene Delivery AffiNANOTECH leverages affinity-based targeting and sophisticated nanoparticle engineering to enable precise and efficient delivery of genetic materials to specific cells and tissues.
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