Gene Therapy: Delivery Methods, Ex-Vivo vs. In-Vivo

Gene Therapy

Gene therapy involves modifying genetic deficiencies at their source. Thus, the process requiring combining non-defective genes to the diseased tissues and organs operate properly. However, gene therapy should be confused with the molecular bandage that fixes the genetic problems automatically.

Gene Therapy Candidate

When choosing candidates for gene therapy, it is important to consider the type of disorder. A genetic disorder is the best for the gene therapy candidate especially those caused by mutations in single genes. The important issues to consider in a candidate include the genes to pursue and the DNA of the patient.

Gene Delivery

Vectors have been defined by the scientist as the genes that deliver DNA into cells and make it function. However, all vectors are not always perfect in their DNA delivery and treating every disorder. A successful vector must target and integrate genes into the right cells. Further, this vector must activate the genes and also avoid any harmful side effects.

Ex-vivo vs. In-vivo

In-vivo is a direct delivery of genes into the patient’s body cells through injection. On the other hand, ex-vivo involves delivering genes into the cells that are not inside the patient’s body. The latter has fewer chances of causing an immune response since no viruses can be transferred to the patient. Also, physicians can test whether the cells are functioning well before they are delivered into the patient's body.

Bone Marrow Transplants

ADA deficiency and Sickle Cell disease are some of the genetic disorders treated with bone marrow transplants.