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腺嘌呤碱基编辑器纠正人胚胎G6PC3基因致病突变
OBJECTIVE: To determine whether the adenine base editor(ABE7.10)can be used to fix harmful mutations in the human G6PC3 gene. METHODS: To investigate the safety of base-edited embryos, off-target analysis by deep sequencing was used to examine the feasibility and editing efficiency of various sgRNA...
Formato: | Online Artículo Texto |
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Lenguaje: | English |
Publicado: |
Editorial office of Chinese Journal of Hematology
2023
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Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10282874/ https://www.ncbi.nlm.nih.gov/pubmed/37357000 http://dx.doi.org/10.3760/cma.j.issn.0253-2727.2023.04.008 |