CRISPR/Cas-based gene editing in therapeutic strategies for beta-thalassemia-Reference-Cited by-同舟云学术

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CRISPR/Cas-based gene editing in therapeutic strategies for beta-thalassemia

Published:2023-10-25 Issue:12 Volume:142 Page:1677-1703
ISSN:0340-6717
Container-title:Human Genetics
language:en
Short-container-title:Hum. Genet.

Author:

Zeng Shujun,Lei Shuangyin,Qu Chao,Wang Yue,Teng Shuzhi,Huang Ping

Funder

National Natural Science Foundation of China

Publisher

Springer Science and Business Media LLC

Subject

Genetics (clinical),Genetics

Link

https://link.springer.com/content/pdf/10.1007/s00439-023-02610-9.pdf

Reference183 articles.

1. Ahmed MH, Ghatge MS, Safo MK (2020) Hemoglobin: structure, function and allostery. Subcell Biochem 94:345–382. https://doi.org/10.1007/978-3-030-41769-7_14

2. Albitar M, Care A, Peschle C, Liebhaber SA (1992) Developmental switching of messenger RNA expression from the human alpha-globin cluster: fetal/adult pattern of theta-globin gene expression. Blood 80:1586–1591

3. Ali S, Mumtaz S, Shakir HA, Khan M, Tahir HM, Mumtaz S, Mughal TA, Hassan A, Kazmi SAR, Sadia IM, Khan MA (2021) Current status of beta-thalassemia and its treatment strategies. Mol Genet Genom Med 9:e1788. https://doi.org/10.1002/mgg3.1788

4. Amendola M, Brusson M, Miccio A (2022) CRISPRthripsis: the risk of CRISPR/Cas9-induced Chromothripsis in gene therapy. Stem Cells Transl Med 11:1003–1009. https://doi.org/10.1093/stcltm/szac064

5. Amit M, Itskovitz-Eldor J (2009) Embryonic stem cells: isolation, characterization and culture. Adv Biochem Eng Biotechnol 114:173–184. https://doi.org/10.1007/10_2008_20

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1. Construction and Stability of All-in-One Adenovirus Vectors Simultaneously Expressing Four and Eight Multiplex Guide RNAs and Cas9 Nickase;International Journal of Molecular Sciences;2024-08-12

2. Regulatory Assessment of Casgevy for the Treatment of Transfusion-Dependent β-Thalassemia and Sickle Cell Disease with Recurrent Vaso-Occlusive Crises;Current Issues in Molecular Biology;2024-07-30

3. CRISPR technology in human diseases;MedComm;2024-07-29

4. Current progress in CRISPR-Cas systems for rare diseases;Progress in Molecular Biology and Translational Science;2024

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