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Short-hairpin RNA against aberrant HBBIVSI-110(G>A) mRNA restores β-globin levels in a novel cell model and acts as mono- and combination therapy for β-thalassemia in primary hematopoietic stem cells.
Patsali P, Papasavva P, Stephanou C, Christou S, Sitarou M, Antoniou MN, Lederer CW, Kleanthous M. Patsali P, et al. Among authors: antoniou mn. Haematologica. 2018 Sep;103(9):e419-e423. doi: 10.3324/haematol.2018.189357. Epub 2018 Apr 26. Haematologica. 2018. PMID: 29700171 Free PMC article. No abstract available.
Correction of IVS I-110(G>A) β-thalassemia by CRISPR/Cas-and TALEN-mediated disruption of aberrant regulatory elements in human hematopoietic stem and progenitor cells.
Patsali P, Turchiano G, Papasavva P, Romito M, Loucari CC, Stephanou C, Christou S, Sitarou M, Mussolino C, Cornu TI, Antoniou MN, Lederer CW, Cathomen T, Kleanthous M. Patsali P, et al. Among authors: antoniou mn. Haematologica. 2019 Nov;104(11):e497-e501. doi: 10.3324/haematol.2018.215178. Epub 2019 Apr 19. Haematologica. 2019. PMID: 31004018 Free PMC article. No abstract available.
Evaluation of Mono- and Bi-Functional GLOBE-Based Vectors for Therapy of β-Thalassemia by HBBAS3 Gene Addition and Mutation-Specific RNA Interference.
Koniali L, Flouri C, Kostopoulou MI, Papaioannou NY, Papasavva PL, Naiisseh B, Stephanou C, Demetriadou A, Sitarou M, Christou S, Antoniou MN, Kleanthous M, Patsali P, Lederer CW. Koniali L, et al. Among authors: antoniou mn. Cells. 2023 Dec 15;12(24):2848. doi: 10.3390/cells12242848. Cells. 2023. PMID: 38132168 Free PMC article.
In Utero Gene Therapy (IUGT) Using GLOBE Lentiviral Vector Phenotypically Corrects the Heterozygous Humanised Mouse Model and Its Progress Can Be Monitored Using MRI Techniques.
Shangaris P, Loukogeorgakis SP, Subramaniam S, Flouri C, Jackson LH, Wang W, Blundell MP, Liu S, Eaton S, Bakhamis N, Ramachandra DL, Maghsoudlou P, Urbani L, Waddington SN, Eddaoudi A, Archer J, Antoniou MN, Stuckey DJ, Schmidt M, Thrasher AJ, Ryan TM, De Coppi P, David AL. Shangaris P, et al. Among authors: antoniou mn. Sci Rep. 2019 Aug 12;9(1):11592. doi: 10.1038/s41598-019-48078-4. Sci Rep. 2019. PMID: 31406195 Free PMC article.
A comparison of intrauterine hemopoietic cell transplantation and lentiviral gene transfer for the correction of severe β-thalassemia in a HbbTh3/+ murine model.
Dighe NM, Tan KW, Tan LG, Shaw SSW, Buckley SMK, Sandikin D, Johana N, Tan YW, Biswas A, Choolani M, Waddington SN, Antoniou MN, Chan JKY, Mattar CNZ. Dighe NM, et al. Among authors: antoniou mn. Exp Hematol. 2018 Jun;62:45-55. doi: 10.1016/j.exphem.2018.03.006. Epub 2018 Mar 29. Exp Hematol. 2018. PMID: 29605545 Free PMC article.
85 results