* This product is for research use only. Not intended for use in the treatment or diagnosis of disease.

Summary

Gene therapy is a new technology that combines modern medicine and molecular biology by introducing the target gene into the patient and achieving the target gene product. Gene therapy as a new approach to disease treatment has already had some successful applications, and scientific breakthroughs will continue to push gene therapy into mainstream medicine.

Gene therapy can work through a variety of mechanisms:

• Replace the disease-causing gene with a healthy copy of the gene.
• Inactivate or turn off disease-causing genes that do not work properly.
• Turn on a gene to help fight the disease.
• Removing a section of DNA that can impair the function of a gene and cause disease.
• Introducing new or modified genes into the body to help treat disease.

A variety of vectors have been developed to carry the target genes into the body, these vectors can be injected directly or intravenously into specific tissues in the body, where it is taken up by individual cells. Alternatively, samples of patient cells can be taken in a laboratory setting and exposed to the carrier. Cells containing the vector are then returned to the patient. If the treatment is successful, the new gene delivered by the carrier will produce a functional protein, or the edited molecule will correct the DNA error and restore protein function.

• Plasmid DNA

Circular DNA molecules can be genetically engineered to carry therapeutic genes into human cells.

• Viral vectors

Viruses have the natural ability to deliver genetic material to cells, so some gene therapy products are derived from viruses (Retroviruses, adenoviruses, adeno-associated viruses, etc). Once a virus has been modified to eliminate its ability to cause infectious disease, these modified viruses can be used as vehicles to carry therapeutic genes into human cells.

• Bacterial vectors

Bacteria can be modified to prevent them from causing infectious diseases and then used as vehicles to carry therapeutic genes into human tissues.

Application

• CAR T cell therapy (or chimeric antigen receptor T cell therapy) is an example of cell-based gene therapy. CAR T cell therapy introduces genes into human T cells, which are a type of immune cell. The gene provides instructions for making a protein, called a chimeric antigen receptor (CAR), that attaches to cancer cells. The modified immune cells can specifically attack cancer cells.
• Adenovirus Gendicine is a gene therapy based on the p53 gene, which is used to treat head and neck cancers.
• Genome editing is a new technique that could be used in gene therapy. Instead of adding new genetic material, genome editing introduces gene-editing tools that can alter existing DNA in cells. Genome editing technology allows the addition, removal, or alteration of genetic material at precise locations in the genome. CRISPR-Cas9 is a well-known type of genome editing.

BOC Sciences' Transfection Kits

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