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Identification of AAV serotypes for gene therapy in Krabbe iPSCs-derived brain organoids.

作者信息

Lv Yafeng, Cui Zhi, Li Hongbo, Wang Jing, Wei Mulan, Hu Yuanlang, Li Xun, Cao Chunyu, Zhang Ye, Wang Wei

机构信息

Hubei Key Laboratory of Tumor Microenvironment and Immunotherapy, College of Basic Medical Sciences, China Three Gorges University, Yichang, Hubei 443000, China.

State Key Laboratory of Medical Molecular Biology, Department of Biochemistry and Molecular Biology, Institute of Basic Medical Sciences, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100005, China.

出版信息

Genes Dis. 2024 Mar 19;12(1):101269. doi: 10.1016/j.gendis.2024.101269. eCollection 2025 Jan.

DOI:10.1016/j.gendis.2024.101269
PMID:39534572
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11555342/
Abstract
摘要
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/2f86/11555342/0ca9dca43ad7/gr1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/2f86/11555342/0ca9dca43ad7/gr1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/2f86/11555342/0ca9dca43ad7/gr1.jpg

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本文引用的文献

1
Advances in the Diagnosis and Treatment of Krabbe Disease.克拉伯病的诊断与治疗进展
Int J Neonatal Screen. 2021 Aug 18;7(3):57. doi: 10.3390/ijns7030057.
2
Current Clinical Applications of In Vivo Gene Therapy with AAVs.腺相关病毒(AAV)体内基因治疗的临床应用现状。
Mol Ther. 2021 Feb 3;29(2):464-488. doi: 10.1016/j.ymthe.2020.12.007. Epub 2020 Dec 10.
3
Generation of a human induced pluripotent stem cell line PUMCi001-A from a patient with Krabbe disease.从一名克拉伯病患者身上生成人诱导多能干细胞系PUMCi001-A。
Stem Cell Res. 2020 Oct;48:101937. doi: 10.1016/j.scr.2020.101937. Epub 2020 Aug 2.
4
Cerebral Organoids: A Human Model for AAV Capsid Selection and Therapeutic Transgene Efficacy in the Brain.脑类器官:用于腺相关病毒衣壳选择及脑内治疗性转基因疗效研究的人类模型
Mol Ther Methods Clin Dev. 2020 Jun 1;18:167-175. doi: 10.1016/j.omtm.2020.05.028. eCollection 2020 Sep 11.
5
Krabbe disease: One Hundred years from the bedside to the bench to the bedside.克拉伯病:从床边到实验室再回到床边的百年历程。
J Neurosci Res. 2016 Nov;94(11):982-9. doi: 10.1002/jnr.23743.