Gene Editing of the Endogenous Cryptic 3′ Splice Site Corrects the RNA Splicing Defect in the β<sup>654</sup>-Thalassemia Mouse Model-Reference-Cited by-同舟云学术

Gene Editing of the Endogenous Cryptic 3′ Splice Site Corrects the RNA Splicing Defect in the β⁶⁵⁴-Thalassemia Mouse Model

Published:2024-08-13 Issue: Volume: Page:
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Lu Dan¹²³^ORCID,Gong Xiuli¹³,Guo Xinbing¹³,Cai Qin¹³,Chen Yanwen¹³,Zhu Yiwen¹³,Sang Xiao¹³,Yang Hua¹³,Xu Miao¹³,Zeng Yitao¹³,Li Dali¹⁴,Zeng Fanyi¹²³⁵^ORCID

Affiliation:

1. Shanghai Institute of Medical Genetics, Shanghai Children’s Hospital, School of Medicine, Shanghai Jiao Tong University, Shanghai, China

2. Department of Histo-Embryology, Genetics and Developmental Biology, Shanghai Jiao Tong University School of Medicine, Shanghai, China

3. NHC Key Laboratory of Medical Embryogenesis and Developmental Molecular Biology & Shanghai Key Laboratory of Embryo and Reproduction Engineering, Shanghai, China

4. Shanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology, Institute of Biomedical Sciences and School of Life Sciences, East China Normal University, Shanghai, China

5. School of Pharmacy, Macau University of Science and Technology, Macau, China.

Publisher

Mary Ann Liebert Inc

Link

https://www.liebertpub.com/doi/pdf/10.1089/hum.2023.202

Reference39 articles.

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4. beta-Thalassemia in Chinese: use of in vivo RNA analysis and oligonucleotide hybridization in systematic characterization of molecular defects.

5. Gene Mutation Spectrum of Thalassemia Among Children in Yunnan Province