MicroRNA (miRNA) is a type of non coding small RNA molecule with a length of approximately 20-25 nucleotides, which can inhibit gene expression after transcription by binding to the mRNA of target genes. This mechanism can regulate a wide range of biological processes, including cell differentiation, proliferation, apoptosis, etc., and therefore plays an important role in development, cancer, cardiovascular disease, and neurodegenerative diseases. Since the discovery of miRNAs, their crucial role in gene regulation has sparked great research interest.

The production and regulatory mechanism of miRNA^【1】

In the field of gene therapy, miRNA, as a natural gene regulatory factor, has shown extensive potential. At present, the application of miRNA in gene therapy mainly focuses on the following aspects:

1. cancer treatment

MiRNAs are involved in the occurrence and development of various cancers. Due to the abnormal expression levels of specific microRNAs in many cancers, such asChronic lymphocytic leukemiaDeletion of miR-15 and miR-16 in CLL. The absence of these miRNAs is considered a key factor in the development of CLL^【2】Scientists are studying the use of miRNA to restore normal gene expression and prevent tumor growth. For example, some tumor suppressor miRNAs can be reintroduced into tumor cells through gene therapy to inhibit cancer cell proliferation and metastasis. On the other hand, some miRNAs that promote tumorigenesis can be specifically suppressed to reduce their pathogenic effects.

2. cardiovascular disease

The application prospects of miRNA in the treatment of cardiovascular diseases have also received widespread attention. For example, miRNA-21 is involved in the regulation of cardiac fibrosis, and inhibiting this miRNA can slow down the progression of heart disease^【3】By regulating miRNAs related to heart function, it is expected to develop more targeted treatment plans to repair damaged heart tissue.

3. Neurodegenerative diseases

In Parkinson's diseaseAlzheimer diseaseIn neurodegenerative diseases such as Alzheimer's disease, miRNA expression is often abnormal. Research has shown that specific miRNAs can regulate the survival and regeneration of neurons. The dysfunction of a range of miRNAs is associated with the deposition of β - amyloid protein, excessive phosphorylation of tau protein, synaptic dysfunction, and Alzheimer's diseaseNeuronal autophagy/Related to phenomena such as apoptosis, these miRNAs have the potential to serve as AD biomarkers^【4】If these miRNAs are manipulated through gene therapy, it may provide new ideas for the treatment of neurological diseases.

Despite the broad potential of miRNA in gene therapy, its effective delivery remains one of the main challenges. MiRNAs need to be stably and efficiently delivered to target cells without degradation in the blood or other internal environments. To address this issue,Scientists are researching various delivery technologies, including liposomes, polymer nanoparticles, exosomes, and more. Nanoparticles, as drug delivery carriers, have become a popular choice due to their small size, strong modifiability, and good targeting abilityhaveThe delivery method of prospects.

1. Personalized medicine

Extracellular vesicles such as exosomes carry biomolecules such as proteins, lipids, RNA, etc., which play an important role in regulating gene expression, tumors, immune regulation, and neurological diseases. The miRNAs carried by them play an important role in cell communication, especially in the tumor microenvironment. MiRNA can reflect the specific state of cells and can therefore serve as a biomarker for diseases, for diagnosis and personalized treatment. By isolating, purifying, analyzing, and characterizing extracellular vesicles from patients' body fluids, doctors can develop targeted treatment plans based on changes in the patient's miRNA expression profile. This provides a new tool for accurate diagnosis and helps clinical doctors make targeted treatment decisions quickly.

2. Nanoparticle delivery

MiRNA, as a natural tool for gene regulation, is considered to be a key factor in gene therapyhaveOne of the potential molecules. Encapsulating specific mRNA in delivery systems such as nanoparticles can ensure stable transmission in vivo and effective delivery to target cells. For example, in cancer treatment, miRNA delivery is used to inhibit tumor growth or restore normal cell function. By combining with nanotechnology, drug delivery systems can significantly enhance the targeting and efficacy of miRNA.Nanoparticle characterization can help to conduct detailed analysis of these nanoparticle carriers, thereby improving the accuracy and effectiveness of treatment. For example, the NanoCoulter nanoparticle analysis system based on the principle of RPS technology can accurately characterize the size, concentration, and potential of these nanoscale carriers such as extracellular vesicles, liposomes, and LNPs, ensuring the uniformity and stability of each batch of carriers^【5,6,7】.

3. Combination therapy

The combination of miRNA with other therapies such as chemotherapy, immunotherapy, etc. may enhance the therapeutic effect. For example, by regulating immune responses or inhibiting tumor proliferation, the sensitivity of chemotherapy drugs can be increased, thereby enhancing therapeutic efficacy.

MiRNA has enormous potential in gene therapy, and its discovery and application have brought new opportunities for precision medicine. Although there are still challenges such as delivery and treatment stability, with the advancement of technology and in-depth research, the application prospects of miRNA in personalized medicine in the future are undoubtedly bright.