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Gene therapy is a technology to correct the defective genes that lead to the development of diseases. Nucleic acid (NA)-based molecules (deoxyribonucleic acid, complementary deoxyribonucleic acid, complete gene, ribonucleic acid and oligonucleotide) are used as research tools in the emerging fields of gene therapy and molecular medicine.
In recent years, NA-based compounds have produced very promising drug candidates for the treatment of various diseases, including cancer, infectious diseases, diabetes, cardiovascular diseases, inflammatory and neurodegenerative diseases, cystic fibrosis, hemophilia and other genetic diseases. However, NA-based drug has some shortcomings which hinder its development, including poor chemical and thermodynamic stability, short in vivo half-life, limited production and high production cost, high in vivo toxicity and side effects, etc.
Fig.1 Schematic illustration of liposome-based antisense-oligonucleotides delivery to the cells
Liposome drug formulations can be used to overcome the non-ideal properties of drugs, such as limited solubility, serum stability, circulation half-life, biodistribution and target tissue selectivity. At present, it has attracted considerable interest to use liposomes as carriers of NAs, either as plasmid vectors for gene therapy, or delivery of smaller NA species. Large unilamellar vesicles (LUVs) are the preferred choice of liposome delivery system for NA drugs because of their favorable drug/lipid ratio and more predictable drug release kinetics. With the improvement of formulation technology, the encapsulation system of cationic liposomes has been widely used as a means to promote NA and intracellular delivery.
Fig.2 Lipid-based carriers for NA delivery
A well-designed liposomal delivery system will be able to reduce the toxicity and increase the efficacy of NA-based drugs by optimizing the delivery of NA to target tissues. The delivery of liposomal NA will depend on the physical and biochemical properties of the liposome, including stability, size, charge, hydrophobicity, interaction with serum proteins and interaction with the surface of non-target cells.
Ideally, liposomes for NA delivery will have the following properties:
(i) They will be safe and well-tolerated;
(ii) They will have appropriate pharmacokinetic properties to ensure delivery to the intended disease site;
(iii) They will mediate effective intracellular delivery of intact NA;
(iv) They will be non-immunogenic, allowing the use of multiple-dose treatment regimens;
(v) They will be stable at the time of manufacture, so mass products with uniform and reproducible specifications can be prepared.
The liposome delivery system used to encapsulate NA usually contains multiple lipid components, each of which plays a role in determining the physical and pharmacological properties of the entire system. At the same time, the pharmacokinetics, biodistribution and intracellular delivery of liposome payloads are also largely dependent on the physical and chemical properties of the carrier.
As a liposome development service provider with more than 10 years of experience, Creative Biolabs promises to provide the best service for every client through reasonable design and comprehensive evaluation in the development process of any kind of liposome formulation. If you are interested in our services, please feel free to contact us.