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Genes are segments of DNA responsible for instructing cells to produce proteins that perform essential functions in our bodies. When genes are defective or mutated they fail to produce normally functioning proteins, leading to missing or defective proteins and the disruption of normal cell function and disease.
One gene therapy approach that can be used is to correct the disease process by introducing a therapeutic gene into patient’s cells. The new gene enables normal protein production—potentially addressing the underlying cause of the disease.
The therapeutic gene is delivered to cells using vectors, or carriers, that are often derived from viruses. The viruses are modified so that they cannot replicate or cause disease. The virus transports the working gene into the cell’s nucleus, where it triggers the production of functioning proteins. This is the approach used by Lysogene.
Gene Therapy FAQ
The content provided here is for informational purposes only and is not intended as medical advice, or as a substitute for the medical advice of a physician.
What is gene therapy?
Gene therapy is an experimental technique that uses genes to treat or prevent disease. By directly inserting therapeutic genes into cells, the new genes then compensate for abnormal or missing genes, enabling cells to function correctly and alleviating disease symptoms. Gene therapies may provide long-term treatment effects after just a single administration.
How does gene therapy work?
Some diseases are caused by mutations in genes that prevent cells from properly producing necessary proteins. Gene therapy works by delivering a healthy copy of a gene to cells, restoring the cells’ ability to produce those proteins. Typically an engineered harmless virus is used to deliver the working copy of a gene into the cells.
How are viruses used in gene therapy?
Because of their great ability to penetrate cells, viruses are typically used in gene therapy to deliver a working copy of a gene into the cells. Viruses contain genetic material (DNA or RNA) surrounded by a protein coat. In gene therapy, engineered, harmless viruses are used as carriers, which are also called vectors, to deliver a therapeutic gene to cells. The virus most commonly used in gene therapy is adeno-associated virus (AAV), as it is safe and can access a broad range of cells.
What diseases can be treated with gene therapy?
Gene therapies are already approved for a range of inherited disorders including eye diseases, neuromuscular disorders, and blood disorders. The blood-brain barrier (BBB) has presented a unique physiological barrier for CNS indications by preventing diffusion of most molecules circulating in the blood. Over the last decade, however, adeno-associated virus (AAV) has emerged as an excellent tool for gene delivery to the CNS. There are two main methods of gene delivery to the CNS: either delivering the therapy directly to the CNS or delivering the therapy in the periphery using vectors able to cross the BBB.