Gene therapy and oligonucleotide drugs can alleviate or cure many diseases that traditional methods cannot cope with, especially those caused by gene defects or abnormalities. Although RNAi is considered to be more effective in treating diseases compared with other methods, there are still some challenges in delivering small interfering RNA (siRNA) to diseased sites for gene therapy. Nanoparticles have unique advantages compared with other carriers and been paid attention to for RNAi delivery.
Exosome is a type of extracellular vesicle (EV) with a diameter of about 30-150 nm. They are secreted by all cell types and can be found in most body fluids, including blood, saliva, and urine. Exosomes are "nanospheres" with bilayer membranes, which contain a wide array of substances from parent cells, such as microRNAs, mRNA expression, lncRNA, DNA, lipids, peptides, and proteins (including oncoprotein, Tumor suppressor genes, transcription regulators and splicing factors). The composition of exosomes is critical because they can be used as biomarkers and provide an indication of their function in biological processes. Learn More
RNA drugs refer to RNA with the function of treating diseases, which can directly regulate the expression of the disease genes from the source, which is one of the effective means of precision medicine. RNA drugs mainly include small interfering RNA, small activated nucleic acids, mRNA drugs, ribozymes, etc. Learn More
Small interfering RNA (siRNA), sometimes called short interfering RNA or silencing RNA, is a type of double-stranded non-coding RNA molecule, usually 20-27 base pairs in length. siRNA can specifically target specific mRNA for degradation and prevent translation, thereby interfering with the expression of specific genes with complementary nucleotide sequences. siRNA can also be used to knock down non-protein-coding genes, such as long non-coding RNA (lncRNA). The use of siRNA can achieve transient silencing in a variety of cell lines, and the experiments are usually limited to a short time range of about 2-4 days.Learn More
mRNA vaccines have the advantages of rapid development, low-cost production, and safe management, and have strong potential to replace conventional vaccines. During the vaccination process, the formulation and delivery strategies of mRNA contribute to the effective expression and presentation of antigens and immune stimulation. mRNA vaccines have been delivered in many forms, including: encapsulated by delivery vectors, such as lipid nanoparticles, polymers, peptides, free mRNA in solution, and wrapped by dendritic cells in vitro. Appropriate delivery materials and formulation methods usually improve vaccine efficacy, which is also affected by the choice of an appropriate route of administration. Learn More
Now, small interfering RNA (siRNA) has become the method of choice for mammalian cell genetic analysis and has the potential to be used as a treatment for a variety of cancers, acquired and inherited diseases. Here, we describe four methods for generating siRNA for mammalian RNAi experiments, each of which has its advantages and disadvantages. The best way to generate siRNA depends on the purpose of the experiment. We will discuss the types of applications that suit them best. Learn More