Introduction to Drug and Gene Delivery

Drug and gene delivery aims to selectively transport therapeutic compounds to specific sites in the body through various medical delivery devices and methods. Traditionally, drugs are administered orally or through injection but targeting specific body parts or tissues poses many challenges. New delivery technologies offer promising solutions for overcoming these challenges.

Nanoparticle-Based Delivery Systems

Nanoparticles are exceptionally useful for drug and gene delivery due to their small size which allows targeting of specific areas. Their large surface area-to-volume ratio enables high drug/gene loading. Various nanoparticles under investigation include:

Liposomes for Drug Delivery

Liposomes are spherical vesicles made of lipid bilayers that can encapsulate both hydrophobic and hydrophilic therapeutic agents. Compared to free drugs, liposome-encapsulated drugs show reduced toxicity, controlled release characteristics and increased efficacy. PEGylation of liposomes helps extend their circulation time in the body. Doxil, the first FDA-approved nanomedicine uses PEGylated liposomes to deliver doxorubicin for cancer treatment with reduced side effects.

Dendrimers: A Precision Delivery Tool

Drug and gene delivery devices Dendrimers are artificially synthesized, highly branched, globular macromolecules that have applications in drug solubilization, encapsulation and targeted delivery. Their well-defined, monodisperse structures allow for reproducible results. Their surface functional groups can be tailored for drug conjugation or targeting ligand attachment. Researchers are developing various polyamidoamine and polypropylene imine dendrimer-based formulations.

Carbon Nanotubes for improved Bioavailability

Carbon nanotubes have shown promise in improving the solubility, stability and bioavailability of hydrophobic drugs. Single-walled carbon nanotubes functionalized with lipid-polyethylene glycol assemblies non-covalently encapsulate paclitaxel, enhancing its delivery to cancer cells in vivo. Studies indicate they could reduce the required dosage and side effects of chemotherapy.

Viral Vectors for Gene Therapy

Viruses have naturally evolved abilities to efficiently deliver genetic cargo into cells making them useful as gene delivery vectors. Commonly used viral vectors in research include adenoviruses, retroviruses, lentiviruses and adeno-associated viruses. While offering high transduction efficiencies, safety issues related to toxicity and immune response limit their clinical use. Researchers work to develop safer, targeted viral vectors.

Non-Viral Delivery Systems

Non-viral vectors offer lower immunogenicity and toxicity compared to viral vectors but generally lower transfection efficiencies. Promising non-viral systems under investigation are cationic lipids and polymers, inorganic nanoparticles and cell-penetrating peptides. Combinations of viral and non-viral components may produce hybrid systems maximizing benefits of both.

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