Gene+Therapy+Final+Activity!

= //GENE THERAPY FINAL ACTIVITY!!!// =

//Below are people decribing their diseases wanting to know whether or not they are eligible for Gene Therapy. If the person is not eligible for gene therapy explain why.//
1. I am blind. A single cell has mutated several genes in the part of my body that would allow my eyes to function. Am I a possible candidate for gene therapy?

2. I am a cystic fibrosis patient. Cystic Fibrosis is an inherited, recessive disorder which means that it can result in more genes. It is a single gene disorder because it only takes place in the CFTR gene on human chromosone 7. If adding a normal copy of the CFTR gene will most likely fix the problem in the affected tissue. Would I be considered a possible candidate for gene therapy?

3. I have Gaucher's Disease. This disease occurs when harmful fatty substances accumulate to excessive levels in the liver, spleen, lungs, bone marrow and can sometimes affect the brain. This is not a single gene disorder and it would take more than one gene replacement to be able to fix my disorder. Would I be a possible candidate for gene therapy?