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Targeting the Apoa1 Locus for Liver-Directed Gene Therapy

Marco De Giorgi,Ang Li,Ayrea Hurley,Mercedes Barzi,Alexandria M. Doerfler,Nikitha A. Cherayil,Harrison E. Smith,Jonathan D. Brown,Charles Y. Lin,Karl-Dimiter Bissig,Gang Bao,William R. Lagor

Molecular Therapy — Methods & Clinical Development（2021）

引用 8|浏览14
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关键词
AAV,CRISPR-Cas9,gene therapy,genome editing,gene targeting,Apoa1,liver,inherited metabolic disorders
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