Cellular and Gene Therapies

Cellular therapy (CT) is the transplantation of human cells to replace or repair damaged tissue and/or cells. With new technologies, innovative products, and limitless imagination, many different types of cells may be used as part of a therapy or treatment for a variety of diseases and conditions.  Some of the cells that may be used include hematopoietic (blood-forming) stem cells (HSC), skeletal muscle stem cells, mesenchymal stem cells, lymphocytes, dendritic cells, and pancreatic islet cells. It is used to treat a variety of blood cancers and hematologic conditions.

Gene therapy is the introduction, removal, or change in the content of a person’s genetic code with the goal of treating a disease, and hereditary diseases in which a defective mutant allele is replaced with a functional one. The transferred genetic material changes how a single protein or group of proteins is produced by the cell. Gene therapy can be used to reduce levels of a disease-causing version of a protein, increase production of disease-fighting proteins, or to produce new/modified proteins. 

 

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