Article
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 - 28 Apr 2023
Muto Valentina, Benigni Federica, Magliocca Valentina, Borghi Rossella, Flex Elisabetta, Pallottini Valentina, Rosa Alessandro, Compagnucci Claudia, Tartaglia Marco
Abstract excerpt
Induced pluripotent stem cells (iPSCs) have been established as a reliable in vitro disease model system and represent a particularly informative tool when animal models are not available or do not recapitulate the human pathophenotype. The recognized limit in using this technology is linked to some degree of variability in the behavior of the individual patient-derived clones. The development of...
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