Gene and Cellular Therapies

Gene therapy is designed to introduce new genetic material into cells to compensate for faulty genes or to make a useful protein. A mutated gene may cause an essential protein to be non-functioning or missing, in that case, gene therapy may be able to introduce a normal copy of the gene, which would produce the a normal and functioning protein. Cell therapy is the administration of whole, living cells in a subject for the treatment of a disorder. Cells used for therapy may be from the same individual (autologous source) or from another individual (allogeneic source). Cells used are obtained from stem cells, or induced pluripotent stem cells (iPSCs), reprogrammed from the dermal fibroblasts or adipocytes. Stem cells or mature cells, differentiated in vitro from stem cells are both used in cell therapy.

  • Somatic cell gene therapy
  • Germline therapy
  • Gene Knockout
  • Stem Cell Therapy

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