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Gene Therapy Methods Based on SPR Technology
The overview of gene therapy
Gene therapy can correct or compensate for diseases caused by defective and abnormal genes by introducing exogenous normal genes into target cells to achieve therapeutic purposes. The way of correction can be to repair the defective gene in situ, or it can be to transfer the functional normal gene to replace the defective gene. Gene therapy mainly treats diseases that seriously threaten human health, including genetic diseases, malignant tumors, cardiovascular diseases, and infectious diseases. Gene therapy is different from conventional treatments. In general, the treatment of disease is aimed at various symptoms caused by gene abnormality, while gene therapy is aimed at the root of the disease - the abnormal gene itself. Gene is the basic functional unit that carries biological genetic information, and it is a specific sequence located on the chromosome. The introduction of foreign genes into biological cells must rely on certain technical methods or vectors. As shown in the figure below, we can see three basic tools for human gene therapy, including adeno-associated virus (AAV) vector, lentiviral vector, and gene editing complex.
Fig.1 Three essential tools for human gene therapy (Dunbar, C, E, et al., 2018)
AAV vector
Adenoviruses are non-enveloped linear double-stranded DNA viruses with a genome length of 36 kb and an inverted terminal repeat region at each end. There are four early transcripts (E1, E2, E3, and E4) distributed on the genome, which are responsible for regulatory functions, and one late transcript is responsible for the coding of structural proteins. The adenovirus vector with the deletion of E1 or E3 gene is generally called the first-generation adenovirus vector. This type of vector can trigger a strong inflammatory response and immune response in the body without purification, and can be used safely after purification. Adenovirus vector is one of the most commonly used viral vectors for gene therapy.
Lentiviral vector
Lentiviral vector is a viral vector derived from human immunodeficiency virus-1 (HIV-1). Lentiviral vectors can effectively integrate foreign genes into the host chromosome, so as to achieve the effect of persistent expression of target sequences. The transfection ability of lentiviral vectors is very strong. For some difficult-to-transfect cells, such as primary cells, stem cells, and undifferentiated cells, the transfection efficiency is greatly improved. The use of lentiviral vectors greatly increases the probability of integrating the target gene into the host cell genome, and can achieve long-term and stable expression of the target gene more conveniently and quickly.
Gene editing complex
Gene editing changes the sequence, expression or function of the target gene by directional modification of the target gene to achieve the addition, deletion and replacement of specific DNA fragments. Gene editing requires the use of the gene editing complex as a scalpel of DNA molecules to achieve the modification of the target gene. One part of the complex can recognize and bind to a specific region of the DNA to be edited, and another part of the complex can perform targeted modification operations on the DNA. The common CRISPR/Cas gene editing complex is the most common DNA molecular scalpel for gene editing.
AAV and lentiviral vectors are the basis for recently approved gene therapies. Gene editing technologies are in translational and clinical stages, but are expected to play an increasing role in the field.
Surface plasmon resonance (SPR) in the process of gene therapy methods research
The AAV vectors, lentiviral vectors, and gene editing technologies mentioned above are all key tools for developing treatments for a variety of diseases, such as sickle cell anemia, Huntington's disease, and certain cancers. To achieve therapeutic efficacy, gene therapy must efficiently and safely deliver nucleic acids to target cells. Accurately characterizing the interaction between viral vectors or gene editing proteins and target cells plays an important role in the research process of improving the safety and efficacy of gene therapy. If you have research needs about gene therapy in your experimental project, our SPR technology platform will be the best analysis tool for your molecular interactions, providing you with high-throughput virus-cell, protein-DNA and protein-cell interaction analysis service. The general process of SPR technology for the research of gene therapy methods is shown in the figure below.
Fig.2 BIAchipTM in the process of gene therapy methods research
According to the tools of gene therapy, on the one hand, it is the research on viral vectors, including the targeting effect of viral vectors, the screening of various modification methods and the release effect of therapeutic genes. Another aspect is the study of the delivery of gene editing proteins. Regarding the research on the above gene therapy methods, first of all, the SPR technology platform can provide high-throughput interaction analysis services, which can increase the reproducibility of experiments and the efficiency of basic screening. By choosing our SPR services, you will greatly save valuable research time and cost, allowing you to devote more energy to more important research. In addition, in the research process of gene therapy methods, the analysis results of various types of interactions need to be very precise. Only in this way can a solid foundation be provided for the subsequent research on gene therapy methods. The SPR technology platform is a highly sensitive and accurate platform, and you can use the data obtained from our SPR technology platform without any worries.
If you are looking for molecular interaction analysis services related to gene therapy, you may consider taking a look at our SPR high-throughput technology platform. If there are other needs related to gene therapy research that we have not mentioned, please feel free to contact us for exclusive customized services. All services are available on a 24/7/365 basis in Creative Proteomics.
Reference
- Dunbar, C. E.; et al. Gene therapy comes of age. Science. 2018, 359(6372): 4672.
For research use only. Not intended for any clinical use.