243 related articles for article (PubMed ID: 35017301)
1. Exon-skipping antisense oligonucleotides for cystic fibrosis therapy.
Kim YJ; Sivetz N; Layne J; Voss DM; Yang L; Zhang Q; Krainer AR
Proc Natl Acad Sci U S A; 2022 Jan; 119(3):. PubMed ID: 35017301
[TBL] [Abstract][Full Text] [Related]
2. Open reading frame correction using splice-switching antisense oligonucleotides for the treatment of cystic fibrosis.
Michaels WE; Pena-Rasgado C; Kotaria R; Bridges RJ; Hastings ML
Proc Natl Acad Sci U S A; 2022 Jan; 119(3):. PubMed ID: 35017302
[No Abstract] [Full Text] [Related]
3. Gene-specific nonsense-mediated mRNA decay targeting for cystic fibrosis therapy.
Kim YJ; Nomakuchi T; Papaleonidopoulou F; Yang L; Zhang Q; Krainer AR
Nat Commun; 2022 May; 13(1):2978. PubMed ID: 35624092
[TBL] [Abstract][Full Text] [Related]
4. Antisense oligonucleotide splicing modulation as a novel Cystic Fibrosis therapeutic approach for the W1282X nonsense mutation.
Oren YS; Avizur-Barchad O; Ozeri-Galai E; Elgrabli R; Schirelman MR; Blinder T; Stampfer CD; Ordan M; Laselva O; Cohen-Cymberknoh M; Kerem E; Bear CE; Kerem B
J Cyst Fibros; 2022 Jul; 21(4):630-636. PubMed ID: 34972649
[TBL] [Abstract][Full Text] [Related]
5. 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]
6. CFTR mRNAs with nonsense codons are degraded by the SMG6-mediated endonucleolytic decay pathway.
Sanderlin EJ; Keenan MM; Mense M; Revenko AS; Monia BP; Guo S; Huang L
Nat Commun; 2022 Apr; 13(1):2344. PubMed ID: 35487895
[TBL] [Abstract][Full Text] [Related]
7. Correction of a Cystic Fibrosis Splicing Mutation by Antisense Oligonucleotides.
Igreja S; Clarke LA; Botelho HM; Marques L; Amaral MD
Hum Mutat; 2016 Feb; 37(2):209-15. PubMed ID: 26553470
[TBL] [Abstract][Full Text] [Related]
8. Antisense Oligonucleotide Therapeutics for Cystic Fibrosis: Recent Developments and Perspectives.
Kim YJ; Krainer AR
Mol Cells; 2023 Jan; 46(1):10-20. PubMed ID: 36697233
[TBL] [Abstract][Full Text] [Related]
9. 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]
10. Antisense oligonucleotide-mediated correction of CFTR splicing improves chloride secretion in cystic fibrosis patient-derived bronchial epithelial cells.
Michaels WE; Bridges RJ; Hastings ML
Nucleic Acids Res; 2020 Jul; 48(13):7454-7467. PubMed ID: 32520327
[TBL] [Abstract][Full Text] [Related]
11. Nonsense-mediated mRNA decay affects nonsense transcript levels and governs response of cystic fibrosis patients to gentamicin.
Linde L; Boelz S; Nissim-Rafinia M; Oren YS; Wilschanski M; Yaacov Y; Virgilis D; Neu-Yilik G; Kulozik AE; Kerem E; Kerem B
J Clin Invest; 2007 Mar; 117(3):683-92. PubMed ID: 17290305
[TBL] [Abstract][Full Text] [Related]
12. The effect of premature termination codon mutations on CFTR mRNA abundance in human nasal epithelium and intestinal organoids: a basis for read-through therapies in cystic fibrosis.
Clarke LA; Awatade NT; Felício VM; Silva IA; Calucho M; Pereira L; Azevedo P; Cavaco J; Barreto C; Bertuzzo C; Gartner S; Beekman J; Amaral MD
Hum Mutat; 2019 Mar; 40(3):326-334. PubMed ID: 30488522
[TBL] [Abstract][Full Text] [Related]
13. Deciphering HERG mutation in long QT syndrome type 2 using antisense oligonucleotide-mediated techniques: Lessons from cystic fibrosis.
Zheng Z; Song Y; Tan X
Heart Rhythm; 2023 Aug; 20(8):1169-1177. PubMed ID: 37121422
[TBL] [Abstract][Full Text] [Related]
14. The suppression of premature termination codons and the repair of splicing mutations in CFTR.
Oren YS; Pranke IM; Kerem B; Sermet-Gaudelus I
Curr Opin Pharmacol; 2017 Jun; 34():125-131. PubMed ID: 29128743
[TBL] [Abstract][Full Text] [Related]
15. The role of common single-nucleotide polymorphisms on exon 9 and exon 12 skipping in nonmutated CFTR alleles.
Steiner B; Truninger K; Sanz J; Schaller A; Gallati S
Hum Mutat; 2004 Aug; 24(2):120-9. PubMed ID: 15241793
[TBL] [Abstract][Full Text] [Related]
16. Capitalizing on the heterogeneous effects of CFTR nonsense and frameshift variants to inform therapeutic strategy for cystic fibrosis.
Sharma N; Evans TA; Pellicore MJ; Davis E; Aksit MA; McCague AF; Joynt AT; Lu Z; Han ST; Anzmann AF; Lam AN; Thaxton A; West N; Merlo C; Gottschalk LB; Raraigh KS; Sosnay PR; Cotton CU; Cutting GR
PLoS Genet; 2018 Nov; 14(11):e1007723. PubMed ID: 30444886
[TBL] [Abstract][Full Text] [Related]
17. A novel exon in the cystic fibrosis transmembrane conductance regulator gene activated by the nonsense mutation E92X in airway epithelial cells of patients with cystic fibrosis.
Will K; Dörk T; Stuhrmann M; Meitinger T; Bertele-Harms R; Tümmler B; Schmidtke J
J Clin Invest; 1994 Apr; 93(4):1852-9. PubMed ID: 7512993
[TBL] [Abstract][Full Text] [Related]
18. Use of adenine base editing and homology-independent targeted integration strategies to correct the cystic fibrosis causing variant, W1282X.
Mention K; Cavusoglu-Doran K; Joynt AT; Santos L; Sanz D; Eastman AC; Merlo C; Langfelder-Schwind E; Scallan MF; Farinha CM; Cutting GR; Sharma N; Harrison PT
Hum Mol Genet; 2023 Nov; 32(23):3237-3248. PubMed ID: 37649273
[TBL] [Abstract][Full Text] [Related]
19. Exon skipping through the creation of a putative exonic splicing silencer as a consequence of the cystic fibrosis mutation R553X.
Aznarez I; Zielenski J; Rommens JM; Blencowe BJ; Tsui LC
J Med Genet; 2007 May; 44(5):341-6. PubMed ID: 17475917
[TBL] [Abstract][Full Text] [Related]
20. Comprehensive Analysis of Combinatorial Pharmacological Treatments to Correct Nonsense Mutations in the CFTR Gene.
Venturini A; Borrelli A; Musante I; Scudieri P; Capurro V; Renda M; Pedemonte N; Galietta LJV
Int J Mol Sci; 2021 Nov; 22(21):. PubMed ID: 34769402
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]
