Gene and Cell Therapy Treatments

Gene and Cell Therapy Treatments

Gene therapy

Many diseases can involve a genetic component a missing gene, multiple copies of a gene, or a gene that is structurally defective. Gene therapy is the process of replacing or modifying genes to correct a deficiency, sometimes by altering the DNA sequence of the target gene or by modifying expression of a gene by turning it off or on. Gene therapy can have an especially profound effect in congenital diseases where the disease is associated with malfunction of a single gene. Researchers are working to identify such genes and develop therapies to replace or modify genes.

Cell therapy

Cell therapy involves replacing or modifying cells to treat disease. Cell therapy can use cells from another person allogenic or the person’s own cells autologous, which may or may not be genetically altered.

Autologous, modified cell therapy is personalized to treat an individual’s condition where scientists take the person’s own cells and genetically change them outside the body, expand the number of modified cells, and then reinfuse these modified cells to produce a therapeutic benefit. Such therapies alter the patient’s cells so their immune system can now target and destroy previously stealth diseased cells.

Examples of cells that are modified to treat disease include T cells commercialized as chimeric antigen receptor T cells CAR T cells, B cells, Natural Killer NK cells, Tumor Infiltrating Lymphocytes TILs, and Dendritic Cells DCs.

Gene editing

Gene editing is the modification of a cell’s DNA with great precision using tools, such as CRISPR based biotechnologies, that enable programmable and targeted repair of damaged gene products.