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

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...

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Detalles Bibliográficos
Autores principales: Muto, Valentina, Benigni, Federica, Magliocca, Valentina, Borghi, Rossella, Flex, Elisabetta, Pallottini, Valentina, Rosa, Alessandro, Compagnucci, Claudia, Tartaglia, Marco
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10178052/
https://www.ncbi.nlm.nih.gov/pubmed/37175696
http://dx.doi.org/10.3390/ijms24097988

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10178052/
https://www.ncbi.nlm.nih.gov/pubmed/37175696
http://dx.doi.org/10.3390/ijms24097988

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