Allele-Specific Disruption of a Common <i>STAT3</i> Autosomal Dominant Allele Is Not Sufficient to Restore Downstream Signaling in Patient-Derived T Cells

Allele-Specific Disruption of a Common <i>STAT3</i> Autosomal Dominant Allele Is Not Sufficient to Restore Downstream Signaling in Patient-Derived T Cells

Dominant negative mutations in the <i>STAT3</i> gene account for autosomal dominant hyper-IgE syndrome (AD-HIES). Patients typically present high IgE serum levels, recurrent infections, and soft tissue abnormalities. While current therapies focus on alleviating the symptoms, hematopoieti...

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Bibliographic Details
Main Authors: Saskia König, Manfred Fliegauf, Manuel Rhiel, Bodo Grimbacher, Tatjana I. Cornu, Toni Cathomen, Claudio Mussolino
Format: Article
Language:English
Published: MDPI AG 2022-10-01
Series:Genes
Subjects:
allele-specific nuclease
gene therapy
CRISPR-Cas
genome editing
primary immunodeficiency
Online Access:https://www.mdpi.com/2073-4425/13/10/1912

Internet

https://www.mdpi.com/2073-4425/13/10/1912

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