Correction of the Marfan Syndrome Pathogenic FBN1 Mutation by Base Editing in Human Cells and Heterozygous Embryos-Reference-Cited by-同舟云学术

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Correction of the Marfan Syndrome Pathogenic FBN1 Mutation by Base Editing in Human Cells and Heterozygous Embryos

Published:2018-11 Issue:11 Volume:26 Page:2631-2637
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Zeng Yanting,Li Jianan,Li Guanglei,Huang Shisheng,Yu Wenxia,Zhang Yu,Chen Dunjin,Chen Jia,Liu Jianqiao,Huang Xingxu

Funder

National Natural Science Foundation of China

Innovation of Science and Technology Commission of Guangzhou, China

China Postdoctoral Science Foundation

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

Reference25 articles.

1. Medical research: next decade’s goals for rare diseases;Austin;Nature,2017

2. Gene therapy comes of age;Dunbar;Science,2018

3. CRISPR-based technologies for the manipulation of eukaryotic genomes;Komor;Cell,2017

4. CRISPR/Cas9-mediated genome editing as a therapeutic approach for Leber congenital amaurosis 10;Ruan;Mol. Ther.,2017

5. One-step biallelic and scarless correction of a β-thalassemia mutation in patient-specific iPSCs without drug selection;Liu;Mol. Ther. Nucleic Acids,2017

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2. The impact of counselors’ values and religious beliefs on their role identity and perspectives on heritable genome editing: a qualitative interview study;Humanities and Social Sciences Communications;2024-08-22

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4. Navigating the CRISPR/Cas Landscape for Enhanced Diagnosis and Treatment of Wilson’s Disease;Cells;2024-07-18

5. An exploration of alternative therapeutic targets for aortic disease in Marfan syndrome;Drug Discovery Today;2024-07

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