Cell-penetrating peptide-conjugated, splice-switching oligonucleotides mitigate the phenotype in BTK/Tec double deficient X-linked agammaglobulinemia model.

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Title: Cell-penetrating peptide-conjugated, splice-switching oligonucleotides mitigate the phenotype in BTK/Tec double deficient X-linked agammaglobulinemia model.
Authors: Bestas B; Department of Laboratory Medicine, Karolinska Institutet, ANA Futura, Alfred Nobels Allé 8 Floor 8 SE-141 52 Huddinge Sweden edvard.smith@ki.se., Estupiñán HY; Department of Laboratory Medicine, Karolinska Institutet, ANA Futura, Alfred Nobels Allé 8 Floor 8 SE-141 52 Huddinge Sweden edvard.smith@ki.se.; Departamento de Ciencias Básicas, Universidad Industrial de Santander Bucaramanga Colombia., Wang Q; Department of Laboratory Medicine, Karolinska Institutet, ANA Futura, Alfred Nobels Allé 8 Floor 8 SE-141 52 Huddinge Sweden edvard.smith@ki.se., Kharazi S; Department of Laboratory Medicine, Karolinska Institutet, ANA Futura, Alfred Nobels Allé 8 Floor 8 SE-141 52 Huddinge Sweden edvard.smith@ki.se., He C; Department of Microbiology, Tumor and Cell Biology, Karolinska Institutet Stockholm Sweden., K Mohammad D; Department of Laboratory Medicine, Karolinska Institutet, ANA Futura, Alfred Nobels Allé 8 Floor 8 SE-141 52 Huddinge Sweden edvard.smith@ki.se., Gupta D; Department of Laboratory Medicine, Karolinska Institutet, ANA Futura, Alfred Nobels Allé 8 Floor 8 SE-141 52 Huddinge Sweden edvard.smith@ki.se.; Department of Paediatrics, University of Oxford Oxford OX3 7TY UK., Wiklander OPB; Department of Laboratory Medicine, Karolinska Institutet, ANA Futura, Alfred Nobels Allé 8 Floor 8 SE-141 52 Huddinge Sweden edvard.smith@ki.se.; Breast Center, Karolinska Comprehensive Cancer Center, Karolinska University Hospital Stockholm Sweden., Lehto T; Department of Laboratory Medicine, Karolinska Institutet, ANA Futura, Alfred Nobels Allé 8 Floor 8 SE-141 52 Huddinge Sweden edvard.smith@ki.se.; Institute of Techology, University of Tartu, Tartu Estonia.; Institute of Pharmacy, Faculty of Medicine, University of Tartu, Nooruse 1, 50411 Tartu Estonia., Lundin KE; Department of Laboratory Medicine, Karolinska Institutet, ANA Futura, Alfred Nobels Allé 8 Floor 8 SE-141 52 Huddinge Sweden edvard.smith@ki.se., Berglöf A; Department of Laboratory Medicine, Karolinska Institutet, ANA Futura, Alfred Nobels Allé 8 Floor 8 SE-141 52 Huddinge Sweden edvard.smith@ki.se., Karlsson MCI; Department of Microbiology, Tumor and Cell Biology, Karolinska Institutet Stockholm Sweden., Abendroth F; Medical Research Council Laboratory of Molecular Biology Cambridge CB2 0QH UK.; Department of Chemistry, University of Marburg Marburg D-35043 Germany., El Andaloussi S; Department of Laboratory Medicine, Karolinska Institutet, ANA Futura, Alfred Nobels Allé 8 Floor 8 SE-141 52 Huddinge Sweden edvard.smith@ki.se.; Karolinska ATMP Center, Karolinska Institutet, Karolinska University Hospital SE-171 76 Stockholm Sweden., Gait MJ; Medical Research Council Laboratory of Molecular Biology Cambridge CB2 0QH UK., Wood MJA; Department of Paediatrics, University of Oxford Oxford OX3 7TY UK., Leumann CJ; Department of Chemistry and Biochemistry, University of Bern, Freiestrasse 3 CH-3012 Bern Switzerland., Stetsenko DA; Institute of Cytology and Genetics, Siberian Branch of the Russian Academy of Sciences, 10 Lavrentiev Ave. Novosibirsk 630090 Russia.; Department of Physics, Novosibirsk State University, 2 Pirogov Str. Novosibirsk 630090 Russia., Månsson R; Department of Laboratory Medicine, Karolinska Institutet, ANA Futura, Alfred Nobels Allé 8 Floor 8 SE-141 52 Huddinge Sweden edvard.smith@ki.se.; Department of Clinical Immunology and Transfusion Medicine, Karolinska University Hospital Stockholm Sweden., Wengel J; Department of Physics, Chemistry and Pharmacy, Biomolecular Nanoscale Engineering Center, University of Southern Denmark Odense Denmark., Zain R; Department of Laboratory Medicine, Karolinska Institutet, ANA Futura, Alfred Nobels Allé 8 Floor 8 SE-141 52 Huddinge Sweden edvard.smith@ki.se.; Karolinska ATMP Center, Karolinska Institutet, Karolinska University Hospital SE-171 76 Stockholm Sweden.; Centre for Rare Diseases, Department of Clinical Genetics and Genomics, Karolinska University Hospital SE-171 76 Stockholm Sweden., Smith CIE; Department of Laboratory Medicine, Karolinska Institutet, ANA Futura, Alfred Nobels Allé 8 Floor 8 SE-141 52 Huddinge Sweden edvard.smith@ki.se.; Karolinska ATMP Center, Karolinska Institutet, Karolinska University Hospital SE-171 76 Stockholm Sweden.
Source: RSC chemical biology [RSC Chem Biol] 2025 Mar 31; Vol. 6 (5), pp. 761-771. Date of Electronic Publication: 2025 Mar 31 (Print Publication: 2025).
Publication Type: Journal Article
Journal Info: Publisher: Royal Society of Chemistry Country of Publication: England NLM ID: 101768727 Publication Model: eCollection Cited Medium: Internet ISSN: 2633-0679 (Electronic) Linking ISSN: 26330679 NLM ISO Abbreviation: RSC Chem Biol Subsets: PubMed not MEDLINE
Database: MEDLINE Ultimate
Description
ISSN:2633-0679
DOI:10.1039/d4cb00312h