194 related articles for article (PubMed ID: 38019847)
41. Targeting G542X CFTR nonsense alleles with ELX-02 restores CFTR function in human-derived intestinal organoids.
Crawford DK; Mullenders J; Pott J; Boj SF; Landskroner-Eiger S; Goddeeris MM
J Cyst Fibros; 2021 May; 20(3):436-442. PubMed ID: 33558100
[TBL] [Abstract][Full Text] [Related]
42.
Zhou ZP; Yang LL; Cao H; Chen ZR; Zhang Y; Wen XY; Hu J
Hum Gene Ther; 2019 Sep; 30(9):1101-1116. PubMed ID: 31099266
[TBL] [Abstract][Full Text] [Related]
43. 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]
44. 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]
45. A helper-dependent adenoviral vector rescues CFTR to wild-type functional levels in cystic fibrosis epithelial cells harbouring class I mutations.
Cao H; Ouyang H; Laselva O; Bartlett C; Zhou ZP; Duan C; Gunawardena T; Avolio J; Bear CE; Gonska T; Hu J; Moraes TJ
Eur Respir J; 2020 Nov; 56(5):. PubMed ID: 32457197
[TBL] [Abstract][Full Text] [Related]
46. Seamless Gene Correction in the Human Cystic Fibrosis Transmembrane Conductance Regulator Locus by Vector Replacement and Vector Insertion Events.
Suzuki S; Chosa K; Barillà C; Yao M; Zuffardi O; Kai H; Shuto T; Suico MA; Kan YW; Sargent RG; Gruenert DC
Front Genome Ed; 2022; 4():843885. PubMed ID: 35465025
[No Abstract] [Full Text] [Related]
47. 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]
48. Correctors and Potentiators Rescue Function of the Truncated W1282X-Cystic Fibrosis Transmembrane Regulator (CFTR) Translation Product.
Haggie PM; Phuan PW; Tan JA; Xu H; Avramescu RG; Perdomo D; Zlock L; Nielson DW; Finkbeiner WE; Lukacs GL; Verkman AS
J Biol Chem; 2017 Jan; 292(3):771-785. PubMed ID: 27895116
[TBL] [Abstract][Full Text] [Related]
49. Identification of the amino acids inserted during suppression of CFTR nonsense mutations and determination of their functional consequences.
Xue X; Mutyam V; Thakerar A; Mobley J; Bridges RJ; Rowe SM; Keeling KM; Bedwell DM
Hum Mol Genet; 2017 Aug; 26(16):3116-3129. PubMed ID: 28575328
[TBL] [Abstract][Full Text] [Related]
50. Augmentation of Cystic Fibrosis Transmembrane Conductance Regulator Function in Human Bronchial Epithelial Cells via SLC6A14-Dependent Amino Acid Uptake. Implications for Treatment of Cystic Fibrosis.
Ahmadi S; Wu YS; Li M; Ip W; Lloyd-Kuzik A; Di Paola M; Du K; Xia S; Lew A; Bozoky Z; Forman-Kay J; Bear CE; Gonska T
Am J Respir Cell Mol Biol; 2019 Dec; 61(6):755-764. PubMed ID: 31189070
[TBL] [Abstract][Full Text] [Related]
51. Nanomolar-potency 'co-potentiator' therapy for cystic fibrosis caused by a defined subset of minimal function CFTR mutants.
Phuan PW; Tan JA; Rivera AA; Zlock L; Nielson DW; Finkbeiner WE; Haggie PM; Verkman AS
Sci Rep; 2019 Nov; 9(1):17640. PubMed ID: 31776420
[TBL] [Abstract][Full Text] [Related]
52. No detectable improvements in cystic fibrosis transmembrane conductance regulator by nasal aminoglycosides in patients with cystic fibrosis with stop mutations.
Clancy JP; Rowe SM; Bebok Z; Aitken ML; Gibson R; Zeitlin P; Berclaz P; Moss R; Knowles MR; Oster RA; Mayer-Hamblett N; Ramsey B
Am J Respir Cell Mol Biol; 2007 Jul; 37(1):57-66. PubMed ID: 17347447
[TBL] [Abstract][Full Text] [Related]
53. Restoration of R117H CFTR folding and function in human airway cells through combination treatment with VX-809 and VX-770.
Gentzsch M; Ren HY; Houck SA; Quinney NL; Cholon DM; Sopha P; Chaudhry IG; Das J; Dokholyan NV; Randell SH; Cyr DM
Am J Physiol Lung Cell Mol Physiol; 2016 Sep; 311(3):L550-9. PubMed ID: 27402691
[TBL] [Abstract][Full Text] [Related]
54. High-Throughput Screening for Modulators of CFTR Activity Based on Genetically Engineered Cystic Fibrosis Disease-Specific iPSCs.
Merkert S; Schubert M; Olmer R; Engels L; Radetzki S; Veltman M; Scholte BJ; Zöllner J; Pedemonte N; Galietta LJV; von Kries JP; Martin U
Stem Cell Reports; 2019 Jun; 12(6):1389-1403. PubMed ID: 31080112
[TBL] [Abstract][Full Text] [Related]
55. Functional Restoration of CFTR Nonsense Mutations in Intestinal Organoids.
de Poel E; Spelier S; Suen SWF; Kruisselbrink E; Graeber SY; Mall MA; Weersink EJM; van der Eerden MM; Koppelman GH; van der Ent CK; Beekman JM
J Cyst Fibros; 2022 Mar; 21(2):246-253. PubMed ID: 34666947
[TBL] [Abstract][Full Text] [Related]
56. Spliceosome-mediated RNA trans-splicing with recombinant adeno-associated virus partially restores cystic fibrosis transmembrane conductance regulator function to polarized human cystic fibrosis airway epithelial cells.
Liu X; Luo M; Zhang LN; Yan Z; Zak R; Ding W; Mansfield SG; Mitchell LG; Engelhardt JF
Hum Gene Ther; 2005 Sep; 16(9):1116-23. PubMed ID: 16149910
[TBL] [Abstract][Full Text] [Related]
57. Respiratory syncytial virus engineered to express the cystic fibrosis transmembrane conductance regulator corrects the bioelectric phenotype of human cystic fibrosis airway epithelium in vitro.
Kwilas AR; Yednak MA; Zhang L; Liesman R; Collins PL; Pickles RJ; Peeples ME
J Virol; 2010 Aug; 84(15):7770-81. PubMed ID: 20504917
[TBL] [Abstract][Full Text] [Related]
58. Robust Stimulation of W1282X-CFTR Channel Activity by a Combination of Allosteric Modulators.
Wang W; Hong JS; Rab A; Sorscher EJ; Kirk KL
PLoS One; 2016; 11(3):e0152232. PubMed ID: 27007499
[TBL] [Abstract][Full Text] [Related]
59. Gentamicin-induced correction of CFTR function in patients with cystic fibrosis and CFTR stop mutations.
Wilschanski M; Yahav Y; Yaacov Y; Blau H; Bentur L; Rivlin J; Aviram M; Bdolah-Abram T; Bebok Z; Shushi L; Kerem B; Kerem E
N Engl J Med; 2003 Oct; 349(15):1433-41. PubMed ID: 14534336
[TBL] [Abstract][Full Text] [Related]
60. Ataluren (PTC124) induces cystic fibrosis transmembrane conductance regulator protein expression and activity in children with nonsense mutation cystic fibrosis.
Sermet-Gaudelus I; Boeck KD; Casimir GJ; Vermeulen F; Leal T; Mogenet A; Roussel D; Fritsch J; Hanssens L; Hirawat S; Miller NL; Constantine S; Reha A; Ajayi T; Elfring GL; Miller LL
Am J Respir Crit Care Med; 2010 Nov; 182(10):1262-72. PubMed ID: 20622033
[TBL] [Abstract][Full Text] [Related]
[Previous] [Next] [New Search]
