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• Drug Screening Study
• Genetic Therapy
• Virus Production

Concept of Gene Therapy

Gene therapy is a biomedical technique in which a normal or therapeutic exogenous gene is inserted into a target cell to repair a defective gene or to replace a defective gene to treat a disease.

The narrow concept refers to the replacement or addition of a defective gene in a patient's body with a gene that has a normal function for treatment.

The broader concept refers to the transfer of certain genetic material into the patient's body so that it is expressed in the patient's body, ultimately resulting in the treatment of a disease.

With the understanding of the mechanism of action of genetic material, we began to consider how to artificially intervene in the process of inheritance and expression. Based on the development of genetic technology, we began to modify and modify genes at the molecular level.

Conventional treatment is generally aimed at various symptoms caused by genetic abnormalities, which can be said to treat the symptoms rather than the root causes, while gene therapy is aimed at the root cause of genetic diseases - the abnormal genes themselves. Gene abnormalities are the essence of genetic diseases, as long as the abnormal genes are treated. Corrected, or replaced the defective gene with the normal gene, then the external symptoms will also be solved.

Classification of Gene Therapy

• According to the target cells
  
  • Germ cell gene therapy
  • Somatic cell gene therapy
    Broadly speaking, germ cell gene therapy targets sperm, eggs and early embryonic cells. Somatic cell therapy is limited to localized cells of the individual. Due to the current immaturity of gene therapy technology and the limited knowledge of genes, it is not known whether the knockout or addition of a gene will result in compromised expression beyond the therapeutic target. In addition, gene modification of germ cells can be passed on to offspring and may even affect the gene pool of the entire human race, as well as involving a range of ethical issues, gene therapy is limited to somatic cells and germ cell gene therapy remains off-limits.

• According to the route of administration
  
  • ex vivo
    It refers to the introduction of a vector containing exogenous genes into the human body's own or allogeneic cells (or xenogeneic cells) in vitro, and after cell expansion in vitro, it is returned to the human body. This approach is relatively classic and safe, and the effect is easier to control, but it has many steps, complex technology, high difficulty, and is not easy to popularize.
  • in vivo
    It is the assembly of an exogenous gene in a specific eukaryotic expression vector and its direct introduction into the body. This vector can be viral or non-viral, or even naked DNA. This route is simple and easy to promote, but it is not yet mature and has several problems such as short duration of efficacy, immune rejection and safety.

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