CRISPR/Cas9 and Piggybac Transposon-Based Conversion of a Pathogenic Biallelic TBCD Variant in a Patient-Derived Ipsc Line Allows Correction of PEBAT-Related Endophenotypes.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES（2023）
关键词
iPSCs,CRISPR/Cas9 technology,gene editing,isogenic controls,neurodevelopmental disorder,TBCD,PEBAT
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