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Gene Therapy

Gene Therapy

Gene therapy, as a new means of disease treatment, brings new hope for the treatment of diseases that cannot be cured by traditional medicine. It mainly uses vectors and other delivery systems to introduce exogenous genes into body cells, or uses gene editing technology to directly realize base substitution, knock-in or knock-out of genes, or infuses the cells of a certain tissue or organ back into the body after modification or modification in vitro by gene editing technology, so as to repair the damaged genes and correct the function of genes, and ultimately to achieve the purpose of treating diseases.

• Strategies For Gene Therapy

The strategies of gene therapy are in vivo gene therapy (in vivo therapy) and ex vivo gene therapy (ex vivo therapy).

• In Vivo Gene Therapy
In vivo gene therapy is to transfer viral or non-viral vectors containing repair gene fragments into the patient's body by local or systemic injection, and the genes are inserted into the patient's chromosomes in an integrated manner for gene repair, or delivered to long-term mitotic or slowly dividing cells in a non-integrated manner to complete the stable expression of DNA outside the chromosomes. The entire editing process of in vivo gene therapy takes place in vivo, and the treatment requires not only accurate and efficient editing methods and tools, but also ensuring that the viral vectors are harmless to the body. The gene transfer process must overcome complex intracellular environments and organizational barriers in order to deliver new genetic information into the target cells, which in turn drives efficient expression of the transferred genes.

• Somatic Gene Therapy
Echo gene therapy is to separate the cells from the patient's body, introduce the genes into the target cells through carriers, localize the genes to one or more loci in the chromosomes of the patient's cells, realize the continuous expression of the transferred genes, and then infuse the modified cells back into the patient's body to achieve the purpose of disease treatment. The genetic modification of the target cells by echo-genotherapy is carried out in laboratories, which has the problem of low editing efficiency. The genetic modification of the target cells in iatrogenic gene therapy is carried out in the laboratory, which has the problem of low editing efficiency. However, the correctly modified cells can be screened out through experiments, and then expanded and cultured, and then infused back into the patient's body to carry out disease treatment after the modified cells reach a certain number. The correctly modified cells must be large enough to reverse the disease, evade immune recognition, survive for a long time, or pass on modified genes to their offspring to maintain normal gene expression in the offspring.

• Vectors For Gene Therapy

• Gene Therapy Vectors
Gene therapy vectors are important media for introducing exogenous genes into target cells in vivo, and their carrying capacity and introduction efficiency seriously affect the therapeutic effect, which can be categorized into viral vectors and non-viral vectors.

• Virus Vector
Viral vectors are used to introduce target genes into recipient cells by means of viruses thereby realizing the treatment of diseases. It has been widely used in cells, animal models and clinical applications. Non-viral vectors are used to realize the purpose of gene transfer through non-viral vector materials.

• Non-Viral Vector
The transduction efficiency of non-viral vectors is lower than that of viral vectors, which can only express the target gene instantaneously. With the improvement of technology, new delivery materials, such as nanomediators, have been introduced, and the new generation of delivery materials will become a better choice for gene therapy.