GENE THERAPY

GENE THERAPY

GENE THERAPY

• Plasmapheresis is used to limit immune system rejection
• Used to reduce or prevent disease

MORE INFO ON GENE THERAPY

• Inactivating a mutated gene that is functioning improperly.
• Introduces a new gene into the body to fight disease

MUTATIONS IN DMD GENE

• Cause Duchenne
• Alter the structure of dystrophin

PROS OF GENE THERAPY

• Only hope of finding a cure for disorders
• Cystic fibrosis, Parkinson's & cancer be passed on to children but wiped out
• Gene therapy when successful can have a number of advantages over drug therapy

CONS OF GENE THERAPY

• Current lack of knowledge on this treatment, unknown safety risks
• Effects of the treatment have been short lived.

WAYS THAT GENE THERAPY HEAL

USED TO TREAT

• Duchenne muscular dystrophy
• Severe combined immune deficiency
• Hemophobia
• Neurodegenartive

DYSTROPHIN

Duchenne Muscular Dystrophy

DMD CAN AFFECT....

• Respiratory function
• Heart
• Learning disabilities
• Pain and sensation