Treating Cystic Fibrosis with stem cell therapies

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Treating Cystic Fibrosis with Stem Cell Therapies

Cystic Fibrosis is a genetic disease that effects mostly people of Scandinavian, British and Irish ancestry. The emigration, in recent centuries, of these peoples has led to Cystic Fibrosis appearing in large numbers across North America and Australasia.

Cystic Fibrosis is a recessive genetic disorder, which means that both parents need to be carriers in order for a child to be born with the disease. Many prospective parents are unaware that they carry the Cystic Fibrosis gene until they have a child with Cystic Fibrosis (CF). The reason for this is that carrying the faulty gene does not appear to adversely effect the health of the carrier, although there is some debate about this as carriers tend to have higher rates of certain disorders (chronic rhinitis for example) as compared to the general population.

Both parents being carriers does not mean that all potential offspring will have CF because carriers will have a pair of genes one faulty and one healthy. This means that the parent has a 50% chance of giving their child the faulty gene resulting in the following odds: chances of a child being a carrier 2 in 4 (50%), chances of child who is CF 1 in four (25%), chances of child not having any faulty CF genes 1 in 4 (25%).

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The odds outlined above are for every birth, therefore it is possible in a family of four children, that all or none of the children have CF or are carriers.

The popular conception of CF is that it is a disease affecting the lungs and although this is true, it may be better viewed as a disease resulting in excessive mucous production which causes damage to the lungs, pancreas, liver, sinuses, and reproductive areas.

One of the major causes of death in the past was the damage to pancreatic function which stopped enzyme production needed to break down the fats in food, resulting in malnutrition and weight loss. CF patients take pancreatic enzymes with their food and some drinks (Coca Cola because of the vegetable fats but not lemonade) in order to digest the fats.

Damage to the Pancreas can also result in the person with CF developing type one diabetes which would necessitate insulin injections along with the daily barrage of antibiotics used to fight lung infections.

Reproductive areas are affected by the excessive mucous including CF males. In the CF male, the Vas Deferens becomes blocked and in the female the excess mucous makes conception difficult.

The most damaging effect of CF is the damage to the lungs by the mucous which itself creates the perfect environment for bacterial infections that can eventually lead to irreversible damage and eventual death. It is the area of lung function that stem cell research offers the greatest hope. Stem cells could be used in the future to rebuild the lungs of CF sufferers using their own stem cells whilst replacing the faulty gene with a normal one.

     
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