Show pageBacklinksCite current pageExport to PDFBack to top This page is read only. You can view the source, but not change it. Ask your administrator if you think this is wrong. **[[Gene]] [[delivery]]** is the process of introducing **foreign genetic material (DNA or RNA)** into a cell to alter its function or produce a therapeutic effect. It's a central technique in **[[gene therapy]]**, **[[vaccine development]]**, **[[biotechnology]]**, and **[[cancer immunotherapy]]**. --- ### 🧬 Objectives of Gene Delivery - **Correct genetic defects** (e.g., cystic fibrosis, muscular dystrophy) - **Deliver therapeutic proteins** (e.g., insulin, clotting factors) - **Reprogram immune cells** (e.g., CAR-T cell therapy) - **Modulate gene expression** (e.g., siRNA or antisense therapies) - **Vaccination** (e.g., mRNA vaccines for COVID-19) --- ### 🚀 Gene Delivery Methods #### 1. **Viral Vectors** - **Adenoviruses**, **lentiviruses**, **adeno-associated viruses (AAVs)** - Efficient delivery, especially to dividing and non-dividing cells - Can integrate into host genome (lentivirus) or remain episomal (AAV) - **Limitations**: immunogenicity, limited cargo capacity, potential for insertional mutagenesis #### 2. **Non-viral Methods** - **Lipid nanoparticles (LNPs)** – used in **mRNA vaccines** - **Electroporation** – electrical pulses to increase membrane permeability - **Gene gun** – shoots DNA-coated particles into cells - **Cell-penetrating peptides (CPPs)** – like the SLE-derived antibody from Chen et al. (2025) - **Polymeric carriers**, dendrimers, or nanocarriers --- ### 🧠 Barriers to Effective Gene Delivery - **Cell membrane** – difficult to penetrate without damaging cells - **Endosomal entrapment** – nucleic acids often get degraded in lysosomes - **Immune system** – may neutralize vectors or cause inflammation - **Target specificity** – ensuring delivery to the right cells/tissues - **Genomic integration risks** – unwanted mutations if integrating vectors are used --- ### 🔬 Emerging Strategies - **Targeted delivery** using ligands or antibodies (e.g., tumor-specific markers) - **mRNA delivery** for transient expression (no genomic integration) - **CRISPR-Cas systems** for gene editing - **Autoantibody-mediated delivery** (like in the Chen et al. study), which bypasses endosomes --- ### 💉 Clinical Applications - **mRNA vaccines**: e.g., Pfizer/BioNTech and Moderna COVID-19 vaccines - **CAR-T therapy**: gene delivery to T cells to express chimeric antigen receptors - **Zolgensma**: AAV-based therapy for spinal muscular atrophy - **Onpattro (patisiran)**: lipid nanoparticle delivering siRNA to the liver --- ### ⚖️ Challenges and Future Directions - Improving **efficiency and safety** - Reducing **immunogenicity** - Enabling **repeated dosing** - Achieving **precise tissue targeting** - Developing **scalable and cost-effective platforms** --- ### 🧾 Summary Gene delivery is the **cornerstone of genetic medicine**, with a wide range of therapeutic and research applications. While viral vectors remain powerful, **non-viral technologies**—especially **lipid nanoparticles** and **novel delivery peptides or antibodies**—are transforming the field by offering safer, more flexible, and more targeted delivery options. --- Would you like a comparison table of delivery methods, or help designing a teaching diagram or research slide on this topic? gene_delivery.txt Last modified: 2025/03/26 05:05by 127.0.0.1