2'-O-methoxyethyl splice-switching oligos correct splicing from IVS2-745 β-thalassemia patient cells restoring HbA production and chain rebalance.
Dong A, Ghiaccio V, Motta I, Guo S, Peralta R, Freier SM, Watt A, Damle S, Ikawa Y, Jarocha D, Chappell M, Stephanou C, Delbini P, Chen C, Christou S, Kleanthous M, Smith-Whitley K, Manwani D, Casu C, Abdulmalik O, Cappellini MD, Rivella S, Breda L.
Dong A, et al. Among authors: delbini p.
Haematologica. 2019 May 1;106(5):1433-1442. doi: 10.3324/haematol.2019.226852.
Haematologica. 2019.
PMID: 32439726
Free PMC article.