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

Petros Patsali, Panayiota Papasavva, Coralea Stephanou, Soteroulla Christou, Maria Sitarou, Michael N. Antoniou, Carsten W. Lederer*, Marina Kleanthous

*Corresponding author for this work

Research output: Contribution to journalLetterpeer-review

12 Citations (Scopus)
Original languageEnglish
Pages (from-to)e419-e423
Issue number9
Publication statusPublished - 31 Aug 2018

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