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Gene Theropy (Diseases (Hemophilia, Sickle cell Disease (Faulty gene: HBB)…
Gene Theropy
Diseases
Hemophilia
Sickle cell Disease (Faulty gene: HBB)
Chronic Granulomatous disorder (CGD)
Type one Diabetes
Severe combined immune defieceny (ADA-SCID)
Cystic Fibrosis
Hemophilia
Huntington's Disease
Parkinson's Disease
Vectors
Typically a virus
Delivers a gene to the cells
Much needed
Attack their hosts and introduce genetic material into the host cell
Have a replication cycle
Typically used to carry out good genes into a human cell
Information
Proteins carry out their jobs in the cells
Not a molecular bandage that will fix every genetic problem
Adds corrected copy of a defective gene
To tell if it works you have to see whether the mutated gene affects the tissue or protein
fixes genetic problems at its source