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CRISPR/Cas9 Correction of a Dominant Cis-Double-variant in COL1A1 Isolated from a Patient with Osteogenesis Imperfecta Increases the Osteogenic Capacity of Induced Pluripotent Stem Cells.

Journal of Bone and Mineral Research(2020)

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关键词
OSTEOGENESIS IMPERFECTA,PEPTIDE NUCLEIC ACID,STEM CELL,CRISPR/CAS9,GENE THERAPY
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