D108 CELL FATE DECISIONS: SENESCENCE, REPAIR, AND REGENERATION: Modeling Zaat-Driven Proteotoxicity Using Crispr-Edited Syngeneic Patient-Ipscs

D108 CELL FATE DECISIONS: SENESCENCE, REPAIR, AND REGENERATION: Modeling Zaat-Driven Proteotoxicity Using Crispr-Edited Syngeneic Patient-Ipscs

Following successful targeting of the Z mutation we have demonstrated the resultant isogenic iPSCs lose the pathognomonic AAT protein retention, which is associated with significant changes in gene expression. Analysis of the gene expression signature resulting from correction of iPSCs derived from...

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Veröffentlicht in:American journal of respiratory and critical care medicine 2017-01, Vol.195
Hauptverfasser: Kaserman, J, Estevez, F Molina, Higgins, M, Mostoslavsky, G, Kotton, D N, Wilson, A
Format: Artikel
Sprache:eng
Schlagworte:
Antibiotics
CRISPR
Gene expression
Liver diseases
Mutation
Patients
Proteins
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Zusammenfassung:Following successful targeting of the Z mutation we have demonstrated the resultant isogenic iPSCs lose the pathognomonic AAT protein retention, which is associated with significant changes in gene expression. Analysis of the gene expression signature resulting from correction of iPSCs derived from patients with specific phenotypes might provide insight into pathways associated with lung or liver disease susceptibility in patients with AATD.
ISSN:1073-449X
1535-4970