Pubblicazioni

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

2023 - INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Muto, Valentina; Benigni, Federica; Magliocca, Valentina; Borghi, Rossella; Flex, Elisabetta; Pallottini, Valentina; Rosa, Alessandro; Compagnucci, Claudia; Tartaglia, Marco