References
- Xu XM, Zhou YQ, Luo GX, et al. The prevalence and spectrum of α and β thalassemia in Guangdong Province: implications for the future health burden and population screening. J Clin Pathol. 2004;57(5):517–522.
- Xiong F, Sun M, Zhang X, et al. Molecular epidemiological survey of hemoglobinopathies in the Guangxi Zhuang autonomous region of southern China. Clin Genet. 2010;78(2):139–148.
- Park IH, Arora N, Huo H, et al. Disease-specific induced pluripotent stem cells. Cell. 2008;134(5):877–886.
- Ou Z, Niu X, He W, et al. The combination of CRISPR/Cas9 and iPSC technologies in the gene therapy of human β-thalassemia in mice. Sci Rep. 2016;6:32463.
- Niu X, He W, Song B, et al. Combining single strand oligodeoxynucleotides and CRISPR/Cas9 to correct gene mutations in β-thalassemia-induced pluripotent stem cells. J Biol Chem. 20165;291(32):16576–16585.
- Yang Y, Zhang X, Yi L, et al. Naïve induced pluripotent stem cells generated from β-thalassemia fibroblasts allow efficient gene correction with CRISPR/Cas9. Stem Cells Transl Med. 2016;5(1):8–19.
- Xu P, Tong Y, Liu XZ, et al. Both TALENs and CRISPR/Cas9 directly target the HBB IVS2-654 (C>T) mutation in β-thalassemia-derived iPSCs. Sci Rep. 2015;5:12065.
- Ma N, Shan Y, Liao B, et al. Factor-induced reprogramming and zinc finger nuclease-aided gene targeting cause different genome instability in β-thalassemia induced pluripotent stem cells (iPSCs). J Biol Chem. 2015;290(19):12079–12089.
- Song B, Fan Y, He W, et al. Improved hematopoietic differentiation efficiency of gene-corrected β-thalassemia induced pluripotent stem cells by CRISPR/Cas9 system. Stem Cells Dev. 2015;24(9):1053–1065.
- Chang CJ, Bouhassira EE. Zinc-finger nuclease-mediated correction of α-thalassemia in iPS cells. Blood. 2012;120(19):3906–3914.
- Fusaki N, Ban H, Nishiyama A, et al. Efficient induction of transgene-free human pluripotent stem cells using a vector based on Sendai virus, an RNA virus that does not integrate into the host genome. Proc Jpn Acad Ser B Phys Biol Sci. 2009;85(8):348–362.
- Schlaeger TM, Daheron L, Brickler TR, et al. A comparison of non-integrating reprogramming methods. Nat Biotechnol. 2015;33(1):58–63.
- Fan Y, Luo Y, Chen X, et al. Generation of human β-thalassemia induced pluripotent stem cells from amniotic fluid cells using a single excisable lentiviral stem cell cassette. J Reprod Dev. 2012;58(4):404–409.
- Trevisan M, Desole G, Costanzi G, et al. Reprogramming methods do not affect gene expression profile of human induced pluripotent stem cells. Int J Mol Sci. 2017;18(1). pii: E206.