Cystic Fibrosis
Cystic Fibrosis is an example of a disease that could benefit from the genetic modification that gene therapy would offer. For this autosomal recessive disease, gene therapy is the only hope for a full cure and possible elimination of the disease completely in the future.
Cystic fibrosis causes abnormality in glands. It mainly affects the respiratory and digestive systems and people with cystic fibrosis have an average life span of 25-30 years old. Most therapies simply ease symptoms; gene therapy could be the cure of the disease since the disease comes from a single gene. It affects the human chromosome 7, so if this defective gene could successfully be replaced than the disease could be cured.
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Cystic Fibrosis being an autosomal recessive gene means that to have the disease, both parents must carry the defective gene. The mother and father do not necessarily have the disease themselves, but they carry the gene. They each have one normal gene and one abnormal gene but they don't show signs of the disease because of the dominance of the normal gene. For the child to have the disease, they must receive the abnormal gene of both parents.
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