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Biomarkers and Precision Therapy for Primary Immunodeficiencies: An In Vitro Study Based on Induced Pluripotent Stem Cells From Patients.

Clinical pharmacology and therapeutics - 1 Aug 2020

Genova Elena, Cavion Federica, Lucafò Marianna, Pelin Marco, Lanzi Gaetana, Masneri Stefania, Ferraro Rosalba Monica, Fazzi Elisa Maria, Orcesi Simona, Decorti Giuliana, Tommasini Alberto, Giliani Silvia, Stocco Gabriele

Abstract excerpt

Ataxia telangiectasia (AT) and Aicardi-Goutières syndrome (AGS) are inherited disorders of immunity with prevalent neurological phenotype. Available treatments are only partially effective, and the prognosis is poor. Induced pluripotent stem cells (iPSCs) are obtained by reprogramming patient somatic cells, preserving the donor individual genetic heritage and creating patient-specific disease models, useful to...

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PMID: 32243572
DOI: 10.1002/cpt.1837

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 6 Sept 2026. Manifest publication-graph-cohort-2026-09-06.1.

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