We have collected information about Cystic Fibrosis Gene Delivery for you. Follow the links to find out details on Cystic Fibrosis Gene Delivery.
https://www.cff.org/Research/Research-Into-the-Disease/Restore-CFTR-Function/Gene-Delivery-for-Cystic-Fibrosis-Therapy/
Gene Delivery for Cystic Fibrosis Therapy. For gene editing and gene replacement therapies to work in cystic fibrosis, specifically engineered DNA or RNA molecules need to get inside the cells of the lung or other organs affected by CF. The process of getting these molecules into cells is referred to as gene delivery.
https://www.ncbi.nlm.nih.gov/pubmed/29236544
Purpose/Aim: Cystic fibrosis (CF) is the most common, fatal recessive genetic disease among the Caucasian population. Gene therapy has the potential to treat CF long term, however physiological barriers can prevent VSV-G pseudotyped lentiviral (LV) vectors from efficiently accessing the relevant receptors on the basolateral membrane of airway epithelial cells.Author: Patricia Cmielewski, Nigel Farrow, Sharnna Devereux, David Parsons, Martin Donnelley
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6545485/
Nov 25, 2018 · Gene therapy for treatment of the cystic fibrosis lung has been an active area of research. Advances made with delivery vehicles for gene therapy can inform development of delivery vehicles for gene editing.Author: Craig A. Hodges, Ronald A. Conlon
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC1134927/
Apr 01, 2005 · Gene therapy holds promise for the treatment of a range of inherited diseases, such as cystic fibrosis. However, efficient delivery and expression of the therapeutic transgene at levels sufficient to result in phenotypic correction of cystic fibrosis pulmonary disease has proved elusive.Author: Tim W. R. Lee, Tim W. R. Lee, David A. Matthews, G. Eric Blair
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6266271/
Nov 07, 2018 · Cystic fibrosis (CF) is an autosomal recessive disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that encodes a cAMP-regulated anion channel.Although CF is a multi-organ system disease, most people with CF die of progressive lung disease that begins early in childhood and is characterized by chronic bacterial infection and …Author: Ashley L. Cooney, Paul B. McCray, Patrick L. Sinn
https://www.ncbi.nlm.nih.gov/pubmed/28270008
Cystic fibrosis (CF) is a monogenic autosomal recessive disorder where the defective gene, the cystic fibrosis transmembrane conductance regulator (CFTR), is well identified. Moreover, the respiratory tract can be targeted through noninvasive aerosolized formulations for inhalation.Author: Ilia Villate-Beitia, Jon Zarate, Gustavo Puras, José Luis Pedraz
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