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. |
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| 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 |
| FullText | Text: Availability: 0 |
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| Header | DbId: mdl DbLabel: MEDLINE Ultimate An: 40171248 AccessLevel: 2 PubType: Academic Journal PubTypeId: academicJournal PreciseRelevancyScore: 0 |
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| Items | – Name: Title Label: Title Group: Ti Data: Cell-penetrating peptide-conjugated, splice-switching oligonucleotides mitigate the phenotype in BTK/Tec double deficient X-linked agammaglobulinemia model. – Name: Author Label: Authors Group: Au Data: <searchLink fieldCode="AU" term="%22Bestas+B%22">Bestas B</searchLink>; Department of Laboratory Medicine, Karolinska Institutet, ANA Futura, Alfred Nobels Allé 8 Floor 8 SE-141 52 Huddinge Sweden edvard.smith@ki.se.<br /><searchLink fieldCode="AU" term="%22Estupiñán+HY%22">Estupiñán HY</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Wang+Q%22">Wang Q</searchLink>; Department of Laboratory Medicine, Karolinska Institutet, ANA Futura, Alfred Nobels Allé 8 Floor 8 SE-141 52 Huddinge Sweden edvard.smith@ki.se.<br /><searchLink fieldCode="AU" term="%22Kharazi+S%22">Kharazi S</searchLink>; Department of Laboratory Medicine, Karolinska Institutet, ANA Futura, Alfred Nobels Allé 8 Floor 8 SE-141 52 Huddinge Sweden edvard.smith@ki.se.<br /><searchLink fieldCode="AU" term="%22He+C%22">He C</searchLink>; Department of Microbiology, Tumor and Cell Biology, Karolinska Institutet Stockholm Sweden.<br /><searchLink fieldCode="AU" term="%22K+Mohammad+D%22">K Mohammad D</searchLink>; Department of Laboratory Medicine, Karolinska Institutet, ANA Futura, Alfred Nobels Allé 8 Floor 8 SE-141 52 Huddinge Sweden edvard.smith@ki.se.<br /><searchLink fieldCode="AU" term="%22Gupta+D%22">Gupta D</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Wiklander+OPB%22">Wiklander OPB</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Lehto+T%22">Lehto T</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Lundin+KE%22">Lundin KE</searchLink>; Department of Laboratory Medicine, Karolinska Institutet, ANA Futura, Alfred Nobels Allé 8 Floor 8 SE-141 52 Huddinge Sweden edvard.smith@ki.se.<br /><searchLink fieldCode="AU" term="%22Berglöf+A%22">Berglöf A</searchLink>; Department of Laboratory Medicine, Karolinska Institutet, ANA Futura, Alfred Nobels Allé 8 Floor 8 SE-141 52 Huddinge Sweden edvard.smith@ki.se.<br /><searchLink fieldCode="AU" term="%22Karlsson+MCI%22">Karlsson MCI</searchLink>; Department of Microbiology, Tumor and Cell Biology, Karolinska Institutet Stockholm Sweden.<br /><searchLink fieldCode="AU" term="%22Abendroth+F%22">Abendroth F</searchLink>; Medical Research Council Laboratory of Molecular Biology Cambridge CB2 0QH UK.; Department of Chemistry, University of Marburg Marburg D-35043 Germany.<br /><searchLink fieldCode="AU" term="%22El+Andaloussi+S%22">El Andaloussi S</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Gait+MJ%22">Gait MJ</searchLink>; Medical Research Council Laboratory of Molecular Biology Cambridge CB2 0QH UK.<br /><searchLink fieldCode="AU" term="%22Wood+MJA%22">Wood MJA</searchLink>; Department of Paediatrics, University of Oxford Oxford OX3 7TY UK.<br /><searchLink fieldCode="AU" term="%22Leumann+CJ%22">Leumann CJ</searchLink>; Department of Chemistry and Biochemistry, University of Bern, Freiestrasse 3 CH-3012 Bern Switzerland.<br /><searchLink fieldCode="AU" term="%22Stetsenko+DA%22">Stetsenko DA</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Månsson+R%22">Månsson R</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Wengel+J%22">Wengel J</searchLink>; Department of Physics, Chemistry and Pharmacy, Biomolecular Nanoscale Engineering Center, University of Southern Denmark Odense Denmark.<br /><searchLink fieldCode="AU" term="%22Zain+R%22">Zain R</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Smith+CIE%22">Smith CIE</searchLink>; 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. – Name: TitleSource Label: Source Group: Src Data: <searchLink fieldCode="JN" term="%22101768727%22">RSC chemical biology</searchLink> [RSC Chem Biol] 2025 Mar 31; Vol. 6 (5), pp. 761-771. <i>Date of Electronic Publication: </i>2025 Mar 31 (<i>Print Publication: </i>2025). – Name: TypePub Label: Publication Type Group: TypPub Data: Journal Article – Name: TitleSource Label: Journal Info Group: Src Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Royal+Society+of+Chemistry%22">Royal Society of Chemistry </searchLink><i>Country of Publication: </i>England <i>NLM ID: </i>101768727 <i>Publication Model: </i>eCollection <i>Cited Medium: </i>Internet <i>ISSN: </i>2633-0679 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2226330679%22">26330679 </searchLink><i>NLM ISO Abbreviation: </i>RSC Chem Biol <i>Subsets: </i>PubMed not MEDLINE |
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| RecordInfo | BibRecord: BibEntity: Identifiers: – Type: doi Value: 10.1039/d4cb00312h Languages: – Code: eng Text: English PhysicalDescription: Pagination: StartPage: 761 Titles: – TitleFull: Cell-penetrating peptide-conjugated, splice-switching oligonucleotides mitigate the phenotype in BTK/Tec double deficient X-linked agammaglobulinemia model. Type: main BibRelationships: HasContributorRelationships: – PersonEntity: Name: NameFull: Bestas B – PersonEntity: Name: NameFull: Estupiñán HY – PersonEntity: Name: NameFull: Wang Q – PersonEntity: Name: NameFull: Kharazi S – PersonEntity: Name: NameFull: He C – PersonEntity: Name: NameFull: K Mohammad D – PersonEntity: Name: NameFull: Gupta D – PersonEntity: Name: NameFull: Wiklander OPB – PersonEntity: Name: NameFull: Lehto T – PersonEntity: Name: NameFull: Lundin KE – PersonEntity: Name: NameFull: Berglöf A – PersonEntity: Name: NameFull: Karlsson MCI – PersonEntity: Name: NameFull: Abendroth F – PersonEntity: Name: NameFull: El Andaloussi S – PersonEntity: Name: NameFull: Gait MJ – PersonEntity: Name: NameFull: Wood MJA – PersonEntity: Name: NameFull: Leumann CJ – PersonEntity: Name: NameFull: Stetsenko DA – PersonEntity: Name: NameFull: Månsson R – PersonEntity: Name: NameFull: Wengel J – PersonEntity: Name: NameFull: Zain R – PersonEntity: Name: NameFull: Smith CIE IsPartOfRelationships: – BibEntity: Dates: – D: 31 M: 03 Text: 2025 Mar 31 Type: published Y: 2025 Identifiers: – Type: issn-electronic Value: 2633-0679 Numbering: – Type: volume Value: 6 – Type: issue Value: 5 Titles: – TitleFull: RSC chemical biology Type: main |
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