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Literature DB >> 32210360

Base editing-mediated splicing correction therapy for spinal muscular atrophy.

Xiang Lin^1,2, Haizhu Chen¹, Ying-Qian Lu¹, Shunyan Hong¹, Xinde Hu³, Yanxia Gao³, Lu-Lu Lai¹, Jin-Jing Li¹, Zishuai Wang⁴, Wenqin Ying³, Lixiang Ma⁵, Ning Wang^1,2, Erwei Zuo⁶, Hui Yang⁷, Wan-Jin Chen^8,9.

Abstract

Entities: Disease

Year: 2020 PMID： 32210360 PMCID： PMC7264321 DOI： 10.1038/s41422-020-0304-y

Source DB: PubMed Journal: Cell Res ISSN： 1001-0602 Impact factor: 25.617

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1 in total

1. Cytosine base editor generates substantial off-target single-nucleotide variants in mouse embryos.

Authors: Erwei Zuo; Yidi Sun; Wu Wei; Tanglong Yuan; Wenqin Ying; Hao Sun; Liyun Yuan; Lars M Steinmetz; Yixue Li; Hui Yang
Journal: Science Date: 2019-02-28 Impact factor: 47.728

1 in total

13 in total

1. Expanding the AtLAS of non-coding RNA functions in the brain.

Authors: Matthew J Regier; Jason D Shepherd
Journal: Cell Res Date: 2020-04 Impact factor: 25.617

Review 2. Advances in gene therapy for neurogenetic diseases: a brief review.

Authors: Ying-Xuan Xie; Wen-Qi Lv; Yi-Kun Chen; Shunyan Hong; Xiang-Ping Yao; Wan-Jin Chen; Miao Zhao
Journal: J Mol Med (Berl) Date: 2021-11-27 Impact factor: 4.599

Review 3. CRISPR Modeling and Correction of Cardiovascular Disease.

Authors: Ning Liu; Eric N Olson
Journal: Circ Res Date: 2022-06-09 Impact factor: 23.213

Review 4. Novel genome-editing-based approaches to treat motor neuron diseases: Promises and challenges.

Authors: Annarita Miccio; Panagiotis Antoniou; Sorana Ciura; Edor Kabashi
Journal: Mol Ther Date: 2021-04-03 Impact factor: 11.454

Review 5. The Potential of CRISPR/Cas9 Gene Editing as a Treatment Strategy for Inherited Diseases.

Authors: Sameh A Abdelnour; Long Xie; Abdallah A Hassanin; Erwei Zuo; Yangqing Lu
Journal: Front Cell Dev Biol Date: 2021-12-15

6. High expression of uracil DNA glycosylase determines C to T substitution in human pluripotent stem cells.

Authors: Ju-Chan Park; Hyeon-Ki Jang; Jumee Kim; Jun Hee Han; Youngri Jung; Keuntae Kim; Sangsu Bae; Hyuk-Jin Cha
Journal: Mol Ther Nucleic Acids Date: 2021-11-29 Impact factor: 8.886

Review 7. Primary Coenzyme Q10 Deficiency-7 and Pathogenic COQ4 Variants: Clinical Presentation, Biochemical Analyses, and Treatment.

Authors: Jieqiong Xie; Jiayang Jiang; Qiwei Guo
Journal: Front Genet Date: 2022-01-26 Impact factor: 4.599