LIVER Publication-derived

Alpha-1-Antitrypsin Deficiency Liver Organoid

Source Gómez-Mariano et al. · Hospital 12 de Octubre, Madrid / University of Cambridge · 10.1007/s12072-019-10007-y

👤 Gómez-Mariano G, Matamala N, Martínez S, Justo I, Marcacuzco A, Jimenez C, Monzón S, Huch M, Janciauskiene S, Martínez-Delgado B ⏱ 10080 min 🧫 Human Adult Liver Biopsy (ZZ, MZ, MM genotypes)

Abstract

Gómez-Mariano et al. etablierten Leberorganoids aus Biopsien von Patienten mit homozygoten ZZ, heterozygoten MZ und normalem MM SERPINA1-Genotyp. Die ZZ-Organoids zeigten intrazelluläre Akkumulation von fehlgefaltetem Z-AAT-Protein (nachweisbar per PAS-D-Färbung und Western Blot) und rekonstituierten damit die hepatische Pathophysiologie des A1AT-Mangels. RNA-seq validierte genotypabhängige Expressionsunterschiede. Grundlagenprotokoll für Gen-Therapie-Ansätze (CRISPR-Korrekturen) bei AATD.

Cell source

Human Adult Liver Biopsy (ZZ, MZ, MM genotypes)

Application

Genetic Disease Modeling

Full SOP

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Attribution

This SOP was authored by Organthis based on the published method in Gómez-Mariano et al.. The originating laboratory holds no rights in this SOP and has not endorsed it unless marked Verified.

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