171 related articles for article (PubMed ID: 10944225)
1. Restoration of hemoglobin A synthesis in erythroid cells from peripheral blood of thalassemic patients.
Lacerra G; Sierakowska H; Carestia C; Fucharoen S; Summerton J; Weller D; Kole R
Proc Natl Acad Sci U S A; 2000 Aug; 97(17):9591-6. PubMed ID: 10944225
[TBL] [Abstract][Full Text] [Related]
2. Restoration of human beta-globin gene expression in murine and human IVS2-654 thalassemic erythroid cells by free uptake of antisense oligonucleotides.
Suwanmanee T; Sierakowska H; Lacerra G; Svasti S; Kirby S; Walsh CE; Fucharoen S; Kole R
Mol Pharmacol; 2002 Sep; 62(3):545-53. PubMed ID: 12181431
[TBL] [Abstract][Full Text] [Related]
3. Repair of a splicing defect in erythroid cells from patients with beta-thalassemia/HbE disorder.
Suwanmanee T; Sierakowska H; Fucharoen S; Kole R
Mol Ther; 2002 Dec; 6(6):718-26. PubMed ID: 12498768
[TBL] [Abstract][Full Text] [Related]
4. Repair of thalassemic human beta-globin mRNA in mammalian cells by antisense oligonucleotides.
Sierakowska H; Sambade MJ; Agrawal S; Kole R
Proc Natl Acad Sci U S A; 1996 Nov; 93(23):12840-4. PubMed ID: 8917506
[TBL] [Abstract][Full Text] [Related]
5. RNA repair restores hemoglobin expression in IVS2-654 thalassemic mice.
Svasti S; Suwanmanee T; Fucharoen S; Moulton HM; Nelson MH; Maeda N; Smithies O; Kole R
Proc Natl Acad Sci U S A; 2009 Jan; 106(4):1205-10. PubMed ID: 19164558
[TBL] [Abstract][Full Text] [Related]
6. Restoration of correct splicing in thalassemic pre-mRNA by antisense oligonucleotides.
Dominski Z; Kole R
Proc Natl Acad Sci U S A; 1993 Sep; 90(18):8673-7. PubMed ID: 8378346
[TBL] [Abstract][Full Text] [Related]
7. High-level expression of hemoglobin A in human thalassemic erythroid progenitor cells following lentiviral vector delivery of an antisense snRNA.
Vacek MM; Ma H; Gemignani F; Lacerra G; Kafri T; Kole R
Blood; 2003 Jan; 101(1):104-11. PubMed ID: 12393543
[TBL] [Abstract][Full Text] [Related]
8. Restoration of correct β
Nualkaew T; Jearawiriyapaisarn N; Hongeng S; Fucharoen S; Kole R; Svasti S
Sci Rep; 2019 May; 9(1):7672. PubMed ID: 31113996
[TBL] [Abstract][Full Text] [Related]
9. Engineered U7 snRNA mediates sustained splicing correction in erythroid cells from β-thalassemia/HbE patients.
Preedagasamzin S; Nualkaew T; Pongrujikorn T; Jinawath N; Kole R; Fucharoen S; Jearawiriyapaisarn N; Svasti S
Biochem Biophys Res Commun; 2018 Apr; 499(1):86-92. PubMed ID: 29550480
[TBL] [Abstract][Full Text] [Related]
10. Correction of aberrant pre-mRNA splicing by antisense oligonucleotides in beta-thalassemia Egyptian patients with IVSI-110 mutation.
El-Beshlawy A; Mostafa A; Youssry I; Gabr H; Mansour IM; El-Tablawy M; Aziz M; Hussein IR
J Pediatr Hematol Oncol; 2008 Apr; 30(4):281-4. PubMed ID: 18391696
[TBL] [Abstract][Full Text] [Related]
11. Reversal of aberrant splicing of beta-thalassemia allele by antisense RNA in vitro and in vivo.
Zeng Y; Gu X; Chen Y; Gong L; Ren Z; Huang S
Chin Med J (Engl); 1999 Feb; 112(2):107-11. PubMed ID: 11593572
[TBL] [Abstract][Full Text] [Related]
12. Engineered U7 Small Nuclear RNA Restores Correct β-Globin Pre-mRNA Splicing in Mouse β
d'Arqom A; Nualkaew T; Jearawiriyapaisarn N; Kole R; Svasti S
Hum Gene Ther; 2021 May; 32(9-10):473-480. PubMed ID: 32977730
[TBL] [Abstract][Full Text] [Related]
13. Temperature-dependent splicing of beta-globin pre-mRNA.
Gemignani F; Sazani P; Morcos P; Kole R
Nucleic Acids Res; 2002 Nov; 30(21):4592-8. PubMed ID: 12409448
[TBL] [Abstract][Full Text] [Related]
14. Enhancement of β-Globin Gene Expression in Thalassemic IVS2-654 Induced Pluripotent Stem Cell-Derived Erythroid Cells by Modified U7 snRNA.
Phanthong P; Borwornpinyo S; Kitiyanant N; Jearawiriyapaisarn N; Nuntakarn L; Saetan J; Nualkaew T; Sa-Ngiamsuntorn K; Anurathapan U; Dinnyes A; Kitiyanant Y; Hongeng S
Stem Cells Transl Med; 2017 Apr; 6(4):1059-1069. PubMed ID: 28213976
[TBL] [Abstract][Full Text] [Related]
15. Restoration of correct splicing of thalassemic beta-globin pre-mRNA by modified U1 snRNAs.
Gorman L; Mercatante DR; Kole R
J Biol Chem; 2000 Nov; 275(46):35914-9. PubMed ID: 10969081
[TBL] [Abstract][Full Text] [Related]
16. [Repair of the splicing defect of thalassemic allele beta IVS-2-654 C-->T in cultured human erythroid cells by antisense RNA].
Gu X; Gong L; Zhang W
Zhonghua Yi Xue Za Zhi; 1997 Nov; 77(11):815-8. PubMed ID: 9772473
[TBL] [Abstract][Full Text] [Related]
17. Stable alteration of pre-mRNA splicing patterns by modified U7 small nuclear RNAs.
Gorman L; Suter D; Emerick V; Schümperli D; Kole R
Proc Natl Acad Sci U S A; 1998 Apr; 95(9):4929-34. PubMed ID: 9560205
[TBL] [Abstract][Full Text] [Related]
18. Sensitivity of splice sites to antisense oligonucleotides in vivo.
Sierakowska H; Sambade MJ; Schümperli D; Kole R
RNA; 1999 Mar; 5(3):369-77. PubMed ID: 10094306
[TBL] [Abstract][Full Text] [Related]
19. Therapeutic hemoglobin levels after gene transfer in β-thalassemia mice and in hematopoietic cells of β-thalassemia and sickle cells disease patients.
Breda L; Casu C; Gardenghi S; Bianchi N; Cartegni L; Narla M; Yazdanbakhsh K; Musso M; Manwani D; Little J; Gardner LB; Kleinert DA; Prus E; Fibach E; Grady RW; Giardina PJ; Gambari R; Rivella S
PLoS One; 2012; 7(3):e32345. PubMed ID: 22479321
[TBL] [Abstract][Full Text] [Related]
20. A combined approach for β-thalassemia based on gene therapy-mediated adult hemoglobin (HbA) production and fetal hemoglobin (HbF) induction.
Zuccato C; Breda L; Salvatori F; Breveglieri G; Gardenghi S; Bianchi N; Brognara E; Lampronti I; Borgatti M; Rivella S; Gambari R
Ann Hematol; 2012 Aug; 91(8):1201-13. PubMed ID: 22460946
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]
