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Gene Therapy for Cystic Fibrosis

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Cystic fibrosis is a genetic disorder that is hereditary. This causes the formation of thick sticky mucus in the lungs and digestive tracks of the being. Mucus formation in the lungs might lead to chronic disease and is a life-threatening disorder.Early diagnosis and treatment can prevent the worsening of the disease. The possible treatments for this disease is use of antibiotics through mouth, veins or breathing treatments, inhalers to open the blocked lungs, DNA enzyme therapy to remove the mucus, oxygen therapy and lung transplantation in aggressive stage.

Long term research has been carried out to treat this disease efficiently. The recent advancement in gene therapy has paved a way to treat cystic fibrosis.  The researchers have succeeded in treating this disease by using viral vectors to carry and deliver healthy genes into the affected cells. In the earlier studies, when a viral vector is used, the immune system destroys the vectors thinking its an invader. So the scientists, in their recent studies used a stronger adeno-associated virus was used so that they can deliver the DNA easily and more efficiently. The animal model being used is pig which has been genetically modified to have cystic fibrosis. If this gene therapy works out and results in successful treatment, then cystic fibrosis will be curable in humans too. So the researchers are working towards this treatment to make it available for patients with cystic fibrosis in the near future.


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