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The thick mucus associated with cystic fibrosis may cause intestinal blockages, leading to nausea and vomiting. Children with cystic fibrosis often face lifelong respiratory issues and require regular treatment.
Cystic fibrosis is a painful genetic disease that can lead to infections in the lungs, pancreas and digestive system.
Cystic fibrosis is a common and painful genetic disease that may lead to infections in the lungs. A child whose cystic fibrosis transmembrane regulator genes are both mutated will have cystic fibrosis.
The most notable benefit of gene therapy for cystic fibrosis is that it has the potential to restore health to many who are suffering with the disease. In addition to gene therapy, those who suffer from cystic fibrosis can consider other treatment alternatives, such as proper nutrition. Inhaled medications, such as mucolytics and antibiotics, can be used to fight bacteria and infections associated with cystic fibrosis.
A major concern is how gene therapy could set the table for genetic engineering in the future. Researchers from the University of Pennsylvania and the Howard Hughes Medical Institute previously showed that an enzyme called Sphingomyelin phosphodiesterase C (SMaseC) produced by the S. SMaseC also suppresses a type of voltage-gated potassium channel, known as the Kv1.3 channel, in immune cells.

To try and counteract the effects of the enzyme, the researchers went on to test a collection of approved drugs and natural products in a chemical library.
Cystic fibrosis is a common and painful genetic disease that can lead to infections in the lungs, pancreas and digestive system. Currently, gene therapy can be used to treat lung problems caused by cystic fibrosis, and scientists hope that in the future technologies in gene therapy will treat other organs affected by the disease. Some question the ethics of gene therapy, believing that scientists shouldn’t interfere with the genetic makeup of an individual.
Researchers are still investigating potential side effects and many clinical trials are still underway. Those with the disease should follow a specially tailored dietary program that involves getting the extra calories that they need to fight infections.
One common antibiotic, azithromycin, has been shown to have special benefits for people with this illness, and it may help maintain and improve lung function. In these experiments, the authors used oocytes from the Xenopus type of frog—that had been genetically modified to express CFTR channels on their cell surface—to measure the effect that SMaseC has on CFTR channels. Suppression of these potassium channels is known to weaken host immunity, which would make it more difficult for the CF patients to recover from lung infections.
They found that tannic acid—a readily available and inexpensive natural product that has been used to treat disease as far back as 1850—stopped SMaseC from having a negative effect on both the CFTR and the Kv1.3 channels. According to the National Genome Research Institute, around 30,000 people in the United States have this condition.

It’s not just cystic fibrosis that has the potential to be cured in this way — gene therapy has the potential to permanently eradicate many diseases.
The slippery slope argument is what a lot of people opposed to gene therapy appear to use -- once a little genetic tampering is deemed normal, what's to prevent people from exploiting the technology to its full potential? They saw that the SMaseC enzyme suppresses CFTR channel activity in these experimentally modified frog oocytes, and also in a human lung cell line. The problems originating from genetic defects in CFTR channels are likely made greater if the enzyme reduces the function of the CFTR channel even further. His team is also working hard to understand the exact mechanism by which tannic acid counters the negative actions of SMaseC. Gene therapy for cystic fibrosis started in 1990 when scientists were able to successfully correct a defective cystic fibrosis transmembrane conductance regulator gene. Since that time, techniques and breakthroughs in gene therapy have occurred but this treatment has both benefits and consequences.

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25.11.2015, author: admin

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