Cystic Fibrosis and Gene Therapy

Cystic Fibrosis and Gene Therapy

The average life span of a person with Cystic Fibrosis is 25-30 years of age. Although the more traditional treatments of this disease are adequate, is there something else that could be even better? Gene therapy is fast becoming one of the more studied aspects of genetics today. Let's take a look at some details of Cystic Fibrosis and gene therapy.

Technical Aspects

Cystic Fibrosis (CF) is the most common fatal genetic disease in the United States today. CF is an autosomal recessive disease that occurs approximately one out of 3,300 live births (Cystic Fibrosis Foundation, 1998). Autosomal means that the gene for CF is not carried on the sex chromosomes and males and females are both afflicted by this disease. Recessive inheritance is when both parents "carry" the abnormal gene in their DNA but they themselves do not show evidence of the disease. The mother and father have one normal gene and one abnormal gene and don't show signs of disease because the normal gene dominates the abnormal gene. To have CF, a child must inherit two abnormal genes, one from each parent. Remember that chromosomes are made up of DNA or deoxyribonucleic acid. DNA is made up of genes, and genes are made up of building blocks called base pairs. The specific gene responsible for CF was identified in 1989 on human chromosome 7 (Pseudomonas Genome Project, 1998). A mutation, or change in the genetic material, resulting in a substitution or loss of one of the base pairs causes the CF gene to be abnormal (Cystic Fibrosis Foundation, 1995).

CF does not affect all people the same way. The basic problem for all patients however is an abnormality in glands, which produce mucus or sweat. The mucus produced by people...

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Cystic Fibrosis Foundation, "Gene Therapy". Obtained from the WWW 10/31/98: http://www.cff.org/genether.htm

Genentech Incorporated, "Gene Therapy-An Overview". Obtained from the WWW 10/31/98: http://www.gene.com/AE/AB/IWT/Gene_Therapy_Overview.html

McClean, Phillip. "Variation in Chromosome Structure". Obtained from the WWW 10/15/98: http://www.ndsu.nodak.edu/instruct/mcclean/plsc431/chromstruct/chrmo2.htm

Pseudomonas Genome Project, "Cystic Fibrosis". Obtained from the WWW 10/31/98: http://www.pseudomonas.com/cystic_fibrosis.html

Cystic Fibrosis Foundation, "An Introduction to Cystic Fibrosis for Patients and Families" in Respiratory Diseases and Disorders Sourcebook". Health Reference Series, V. 6, 1995. Sect. 5.1, p. 240.

WWWebster Dictionary. Obtained from the WWW 11/2/98: http://www.m-w.com/