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
Valentina Muto(Bambino Gesù Children's Hospital), Marco Tartaglia(Bambino Gesù Children's Hospital)
Cited by 4
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