2'-O-methoxyethyl splice-switching oligos correct splicing from IVS2-745 β-thalassemia patient cells restoring HbA production and chain rebalance
2'-O-甲氧基乙基剪接转换寡核苷酸可纠正IVS2-745 β-地中海贫血患者细胞的剪接，从而恢复HbA的生成和链的重新平衡。
期刊:Haematologica
影响因子:8.2
doi:10.3324/haematol.2019.226852
Alisa Dong ,Valentina Ghiaccio ,Irene Motta ,Shuling Guo ,Raechel Peralta ,Susan M Freier ,Andy Watt ,Sagar Damle ,Yasuhiro Ikawa ,Danuta Jarocha ,Maxwell Chappell ,Coralea Stephanou ,Paola Delbini ,Connie Chen ,Soteroula Christou ,Marina Kleanthous ,Kim Smith-Whitley ,Deepa Manwani ,Carla Casu ,Osheiza Abdulmalik ,Maria Domenica Cappellini ,Stefano Rivella ,Laura Breda
