What is the Difference Between Ex Vivo and In Vivo Gene Therapy?

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Ex vivo and in vivo gene therapy are two main methods of delivering gene therapy to treat or cure certain medical conditions. The key difference between the two lies in the delivery method:

  • Ex vivo gene therapy involves removing specific cells from a person, genetically altering them in a laboratory, and then transplanting them back into the person. This process typically involves genetically modifying a patient's stem cells, which then replace target cells that have a missing or malfunctioning gene. Ex vivo gene therapy is most frequently applied to hematopoietic stem cells (HSCs) to treat blood and immunological diseases, as well as genetic diseases that affect various organs.
  • In vivo gene therapy refers to the direct delivery of genetic material into the patient's body. New genetic material is first created in a lab and then delivered to the patient's cells directly, often using viral vectors as a delivery system. In vivo gene therapy works by introducing therapeutic genes into the patient's body, sometimes injecting the genetic material directly into a target organ. This method is suitable for treating conditions that affect specific cells, such as cells in the eye.

Both ex vivo and in vivo gene therapy methods have risks and side effects associated with them, and it is essential to discuss with a healthcare provider whether gene therapy is the right choice for a specific medical condition. Scientists continue to study and refine these methods to improve their effectiveness and safety.

Comparative Table: Ex Vivo vs In Vivo Gene Therapy

The principal difference between ex vivo and in vivo gene therapy lies in the delivery method of the therapeutic genes:

Ex Vivo Gene Therapy In Vivo Gene Therapy
Cells are removed from the patient's body, modified in a lab, and then returned to the patient Therapeutic genes are introduced directly into the patient's body
Cells with defective genes are isolated, grown in cultures, and then therapeutic genes are inserted using vectors New genetic material is created in a lab and inserted into the patient's cells directly
Ex vivo gene therapy is applied to certain cell types or selected tissues, such as bone marrow cells In vivo gene therapy is less selective and can be applied to a wider range of cells and tissues
Examples include ex vivo gene insertion using CAR-T and ex vivo gene transfer Examples include gene therapy using viruses or other methods to deliver genes directly into cells

Both methods of gene therapy are being studied to treat various medical conditions, such as blood disorders, inherited diseases, and infections. The choice between ex vivo and in vivo gene therapy depends on the specific research goals and the type of cells or tissues being targeted for treatment.