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Titel:

Rescue of STAT3 Function in Hyper-IgE Syndrome Using Adenine Base Editing.

Dokumenttyp:
Article; Journal Article; Research Support, Non-U.S. Gov't
Autor(en):
Eberherr, Andreas C; Maaske, Andre; Wolf, Christine; Giesert, Florian; Berutti, Riccardo; Rusha, Ejona; Pertek, Anna; Kastlmeier, Miriam T; Voss, Carola; Plummer, Michelle; Sayed, Amina; Graf, Elisabeth; Effner, Renate; Volz, Thomas; Drukker, Micha; Strom, Tim M; Meitinger, Thomas; Stoeger, Tobias; Buyx, Alena M; Hagl, Beate; Renner, Ellen D
Abstract:
STAT3-hyper IgE syndrome (STAT3-HIES) is a primary immunodeficiency presenting with destructive lung disease along with other symptoms. CRISPR-Cas9-mediated adenine base editors (ABEs) have the potential to correct one of the most common STAT3-HIES causing heterozygous STAT3 mutations (c.1144C>T/p.R382W). As a proof-of-concept, we successfully applied ABEs to correct STAT3 p.R382W in patient fibroblasts and induced pluripotent stem cells (iPSCs). Treated primary STAT3-HIES patient fibroblasts sh...     »
Zeitschriftentitel:
CRISPR J
Jahr:
2021
Band / Volume:
4
Heft / Issue:
2
Seitenangaben Beitrag:
178-190
Volltext / DOI:
doi:10.1089/crispr.2020.0111
PubMed:
http://view.ncbi.nlm.nih.gov/pubmed/33876960
Print-ISSN:
2573-1599
TUM Einrichtung:
Institut für Geschichte und Ethik der Medizin; Institut für Humangenetik; Klinik und Poliklinik für Dermatologie und Allergologie; Klinik und Poliklinik für Kinder- und Jugendmedizin
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