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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.
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.
Safer, silencing-resistant lentiviral vectors: optimization of the ubiquitous chromatin-opening element through elimination of aberrant splicing.
Knight S, Zhang F, Mueller-Kuller U, Bokhoven M, Gupta A, Broughton T, Sha S, Antoniou MN, Brendel C, Grez M, Thrasher AJ, Collins M, Takeuchi Y. Knight S, et al. Among authors: antoniou mn. J Virol. 2012 Sep;86(17):9088-95. doi: 10.1128/JVI.00485-12. Epub 2012 Jun 13. J Virol. 2012. PMID: 22696657 Free PMC article.
80 results