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6. Nonsense-mediated RNA Decay Pathway Inhibition Restores Expression and Function of W1282X CFTR. Keenan MM; Huang L; Jordan NJ; Wong E; Cheng Y; Valley HC; Mahiou J; Liang F; Bihler H; Mense M; Guo S; Monia BP Am J Respir Cell Mol Biol; 2019 Sep; 61(3):290-300. PubMed ID: 30836009 [TBL] [Abstract][Full Text] [Related]
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10. Partial correction of endogenous DeltaF508 CFTR in human cystic fibrosis airway epithelia by spliceosome-mediated RNA trans-splicing. Liu X; Jiang Q; Mansfield SG; Puttaraju M; Zhang Y; Zhou W; Cohn JA; Garcia-Blanco MA; Mitchell LG; Engelhardt JF Nat Biotechnol; 2002 Jan; 20(1):47-52. PubMed ID: 11753361 [TBL] [Abstract][Full Text] [Related]
11. Novel Correctors and Potentiators Enhance Translational Readthrough in CFTR Nonsense Mutations. Mutyam V; Sharma J; Li Y; Peng N; Chen J; Tang LP; Falk Libby E; Singh AK; Conrath K; Rowe SM Am J Respir Cell Mol Biol; 2021 May; 64(5):604-616. PubMed ID: 33616476 [TBL] [Abstract][Full Text] [Related]
12. Targeted Integration of a Super-Exon into the CFTR Locus Leads to Functional Correction of a Cystic Fibrosis Cell Line Model. Bednarski C; Tomczak K; Vom Hövel B; Weber WM; Cathomen T PLoS One; 2016; 11(8):e0161072. PubMed ID: 27526025 [TBL] [Abstract][Full Text] [Related]
13. Functional rescue of c.3846G>A (W1282X) in patient-derived nasal cultures achieved by inhibition of nonsense mediated decay and protein modulators with complementary mechanisms of action. Laselva O; Eckford PD; Bartlett C; Ouyang H; Gunawardena TN; Gonska T; Moraes TJ; Bear CE J Cyst Fibros; 2020 Sep; 19(5):717-727. PubMed ID: 31831337 [TBL] [Abstract][Full Text] [Related]
14. Correction of aberrant splicing of the cystic fibrosis transmembrane conductance regulator (CFTR) gene by antisense oligonucleotides. Friedman KJ; Kole J; Cohn JA; Knowles MR; Silverman LM; Kole R J Biol Chem; 1999 Dec; 274(51):36193-9. PubMed ID: 10593905 [TBL] [Abstract][Full Text] [Related]
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