How do You Get Cystic Fibrosis?

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Cystic Fibrosis is an inherited disease that produces a thick and sticky fluid which causes pain with constipation, coughing, weight loss and recurring infections such as pneumonia or sinusitis. You can find more information here: healthline.com/adamcontent/cystic-fibrosis?utm_medium=ask&utm_source=smart&utm_campaign=article_toc&utm_term=Cystic+fibrosis+risk+factor#causesincidenceandriskfactors&ask_return=Cystic+Fibrosiscausesincidenceandriskfactors.

128 With the help of the Cystic Fibrosis Trust, which has a league of highly professional gene therapists, both somatic and Adeno-associated viral vector have made advances. The Adenoviridae, or more commonly known as the cold virus, is genetically altered, allowing the CFTR gene to enter lung cells. A number of small molecules that aim at compensating various mutations of the CFTR gene are under development.

One approach is to develop drugs that get the ribosome to overcome the stop codon and synthesize a full-length CFTR protein. About 10% of CF result from a premature stop codon in the DNA, leading to early termination of protein synthesis and truncated proteins. These drugs target nonsense mutations such as G542X, which consists of the amino acid glycine in position 542 being replaced by a stop codon.

Aminoglycoside antibiotics interfere with DNA synthesis and error-correction. In some cases, they can cause the cell to overcome the stop codon, insert a random amino acid, and express a full-length protein.

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