Direct delivery of the cystic fibrosis transmembrane conductance regulator (CFTR) gene to airway epithelia may offer advantages… Advantages of mRNA include the cytoplasmic location of the translational machinery, pre-empting the need for nuclear uptake, which is … "Cystic fibrosis hope as new gene therapy improves condition," The Daily Telegraph reports. Traditional treatment of cystic fibrosis involves using antibiotics that treat lung … ADVANTAGES Can be used to eradicate disease for future generations=>germline therapy enables the correct gene to be passed down to future generation Can possibly prevent and eliminate hereditary diseases such as cystic fibrosis … Cystic fibrosis is a genetic condition caused by a mutated gene … Cystic fibrosis (CF) is a disease characterized by airway infection, inflammation, remodeling, and obstruction that gradually destroy the lungs. Researchers have, for the first time, managed to successfully "smuggle" healthy copies of genes into the lungs of people with cystic fibrosis. Advantages of Gene Therapy. 2. Summary During the past decade, newborn screening for cystic fibrosis … The main advantage of gene therapy approach is that it deals with the root cause of the disease. Educational aims 1. Developing novel approaches to gene therapy cystic fibrosis. To understand the drawbacks specifically associated with newborn screening for cystic fibrosis and how these can be handled or avoided. To give an insight into the arguments that have led to the implementation of newborn screening for cystic fibrosis in routine screening programmes.

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