Page 222 - 2021_05-Haematologica-web
P. 222
A. Dong et al.
24.Deng W, Rupon JW, Krivega I, et al.
Reactivation of developmentally silenced globin genes by forced chromatin looping. Cell. 2014;158(4):849-860.
25. Breda L, Motta I, Lourenco S, Gemmo C, et al. Forced chromatin looping raises fetal hemoglobin in adult sickle cells to higher levels than pharmacologic inducers. Blood. 2016;128(8):1139-1143.
26.Breda L, Casu C, Gardenghi S, et al. Therapeutic hemoglobin levels after gene transfer in beta-thalassemia mice and in hematopoietic cells of beta-thalassemia
and sickle cells disease patients. PLoS One.
2012;7(3):e32345.
27. Gemignani F, Sazani P, Morcos P, Kole R.
Temperature-dependent splicing of beta- globin pre-mRNA. Nucleic Acids Res. 2002; 30(21):4592-4598.
28. Disterer P, Kryczka A, Liu Y, et al. Development of therapeutic splice-switch- ing oligonucleotides. Hum Gene Ther. 2014;25(7):587-598.
29. Gonzalez-Redondo JM, Kutlar F, Kutlar A, et al. Hb S(C)-beta+-thalassaemia: differ- ent mutations are associated with different
levels of normal Hb A. Br J Haematol.
1988; 70(1):85-89.
30. Crooke ST, Baker BF, Xia S, et al. Integrated
assessment of the clinical performance of GalNAc3-conjugated 2'-O-methoxyethyl chimeric antisense oligonucleotides: I. human volunteer experience. Nucleic Acid Ther. 2019;29(1):16-32.
31.Ammala C, Drury WJ 3rd, Knerr L, et al. Targeted delivery of antisense oligonu- cleotides to pancreatic beta-cells. Sci Adv. 2018;4(10):eaat3386.
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