In vivo genome editing at the albumin locus to treat methylmalonic acidemia

标题
In vivo genome editing at the albumin locus to treat methylmalonic acidemia
作者
关键词
adeno-associated virus, albumin, CRISPR-Cas9, metabolic disorders, methylmalonic acidemia, genome editing
出版物
Molecular Therapy-Methods & Clinical Development
Volume 23, Issue -, Pages 619-632
出版商
Elsevier BV
发表日期
2021-11-12
DOI
10.1016/j.omtm.2021.11.004

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