Gene therapy

• Gene therapy

  = Altering a persons genetic material to treat or cure disease.

  • Replace a faulty gene or inactivate a faulty gene or insert a new gene.
  • Vectors can be viral or non-viral.
  • Liposomes
• Somatic gene therapy

  • Body cells are targeted
  • Changes in genetic material are targeted to specific cells so wont be inherited (doesn't target gametes)
  • Short lived changes (cells dies) so need to use stem cells so they can divide.
• Ex-vivo

  • Blood or bone marrow cells extracted
  • Extracted cells are exposed to virus which inserts the gene into the cells.
  • Cells are grown in lab and returned to person by injection into a vein.
• In-vivo

  • New gene is inserted via a vector into cells inside the body.
• Germ line therapy

  • Potential for new genetic material to be inserted into germ cells (gametes)
  • Illegal- any changes made to the genetic material of those cells is permanent so would be inherited