[180] Chemical modified mRNA therapy for Cystic Fibrosis by targeting Lung cells.

Autor:	Sahu, Itishri, Haque, A. K. M. Ashiqul, Schweizer, Georg R, Weidensee, Brian, Weinmann, Petra, Loretz, Brigitta, Lehr, Claus Michael, Michel, Tatjana, Wendel, Hans Peter
Předmět:	CYSTIC fibrosis PULMONARY fibrosis MESSENGER RNA CELLS CYSTIC fibrosis transmembrane conductance regulator
Zdroj:	COPD: Journal of Chronic Obstructive Pulmonary Disease; Apr2019, Vol. 16 Issue 2, p209-210, 2p
Abstrakt:	Highlights from the article: Gene deficiency diseases where there is lack of functional protein, have found a potential cure in the form of mRNA therapy ([1]) but major challenges for achieving higher bioavailability concerning mRNA based therapy are higher rate of degradation, reduced ability to cross cellular barriers and immunogenicity ([2]) The advances in the field of nano-carriers and chemical modifications of mRNA bases and codon optimization have brought the mRNA based technology into therapeutics race; however, much more has to be achieved ([3], [4]). Additionally, in the light of recent identification of Lung stem cells (Myoepithelial cells and Bronchio Alveolar Stem Cells) ([[13]]), gene correction via sgRNA/Cas9 system could be also fruitful to target and repair stem cells.
Databáze:	Complementary Index
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