Disease Hemophilia Severe Combined Immune Deficiency

(ADA-SCID) or The Bubble Boy Disease.

Description Genetic disease that is characterized by inability of the patient to stimulate blood clotting process in cases of internal or external injuries. It is a genetic disorder that children born without an effective immune system.
Treatment using gene therapy. The disorder can be corrected by introduction of therapeutic gene into the lever of the patient. The therapeutic gene induces the patient to synthesis clotting factors and able to respond normally to both internal and external. ADA-SCID can be treated by bone marrow transfer. An ADA is inserted in the bone marrow cells of the patient. The genetically corrected are then transplanted back into patient’s body then the defective immune system is restored.


Similarities and differences of Somatic and Gene Line Therapy.

  Somatic Cell Therapy Gene line Therapy
  • It is purposed to eliminate the clinical consequences of the disease.
  • The inserted gene is not passed to the patient’s offspring.
  • Corrected gene is into the fertilized egg of an animal with disorder.
  • The gametes gain new gene thus transferred to the next generation.
  • It is important for insertion of a functional gene to correct a genetic disorder.
  • It is important for insertion of functional gene to correct a genetic disorder.