Research has found cystic fibrosis (CF) treatment potential in improved gene therapy. Cystic fibrosis is a hereditary disease that causes the body to produce thick and sticky mucus, which can clog the lungs and obstruct the pancreas. An estimated 30,000 children are affected by cystic fibrosis within the U.S. and roughly 1,000 new cases are diagnosed each year.
Individuals with cystic fibrosis tend to have a shorter than normal life expectancy, but with medical advancements, many of them can live past the age of 40.
Cystic fibrosis primarily affects the lungs and digestive system and is caused by a defective gene. Researchers have found that improving gene therapy could offer hope in the treatment of cystic fibrosis.
Cystic fibrosis is caused by mutations in the CFTR gene. Professor Zeger Debyser from the new study explained, “A few years ago, a new drug was launched that can repair dysfunctional chloride channels. Unfortunately, this medicine only works in a minority of CF patients. As for the impact of gene therapy, previous studies suggested that the treatment is safe, but largely ineffective for cystic fibrosis patients. However, as gene therapy has recently proven successful for disorders such as hemophilia and congenital blindness, we wanted to re-examine its potential for cystic fibrosis.”
The researchers decided to improve the current gene therapy based on inserting the genetic material for chloride channels into the genome of a recombinant AAV viral vector.
Dr. Debyser concluded, “We must not give CF patients false hope. Developing a treatment based on gene therapy will take years of work. For one thing, our study did not involve actual human beings, only mice and patient-derived cell cultures. Furthermore, we still have to examine how long the therapy works. Repeated doses might be necessary. But gene therapy clearly is a promising candidate for further research towards a cure for cystic fibrosis.”
A defect in the gene CFTR leads to unusually thick and sticky mucus that is difficult to cough up out of the lungs. In the digestive tract this mucus impedes on normal pancreatic function, which inhibits food from being broken down and can lead to malnutrition.
If a person only has one of the defective genes, they are carriers of cystic fibrosis and although they don’t have it themselves, they have the potential to pass it on if their partner also has one of the defective genes. For a child to inherit cystic fibrosis both parents must posses the defective gene.
Signs and symptoms of cystic fibrosis include:
Treatment plans are geared towards the child’s specific needs and health problems. Respiratory and digestive therapy will most likely be ongoing forms of treatment as those two areas of the body are greatly affected by cystic fibrosis. Antibiotics are also required, along with regular check-ups, home treatment like postural drainage, and taking the necessary steps to prevent infection.
Respiratory therapy works to slow down lung damage and improve breathing. This form of treatment works to rid the body of excess mucus and keep the lungs healthy.
Digestive therapy involves the use of digestive enzymes to help assist absorption of nutrients in order to prevent malnutrition. Nutritional therapy is also used to replace any lost nutrients. Vitamins, high-calorie food, high-fat foods and drinking nutritional beverages are all ways to utilize nutritional therapy. Lastly, stool softeners may be used to help prevent stool blockages and constipation.
Home remedies for cystic fibrosis involve the following:
It is recommended that you work closely with a doctor, so they can create at home treatment plans to help provide care and treatment if you have cystic fibrosis.
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