Short-hairpin RNA against aberrant HBB[IVSI-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.

Short-hairpin RNA against aberrant HBB[IVSI-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. Haematologica. 2018 Apr 26;: Authors: Patsali P, Papasavva P, Stephanou C, Christou S, Sitarou M, Antoniou MN, Lederer CW, Kleanthous M PMID: 29700171 [PubMed - as supplied by publisher]

https://www.ncbi.nlm.nih.gov/pubmed/29700171?dopt=Abstract

Source: Haematologica - April 26, 2018 Category: Hematology Authors: Patsali P, Papasavva P, Stephanou C, Christou S, Sitarou M, Antoniou MN, Lederer CW, Kleanthous M Tags: Haematologica Source Type: research

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