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Page 1
Biallelic human SHARPIN loss of function induces autoinflammation and immunodeficiency.
Oda H, Manthiram K, Chavan PP, Rieser E, Veli Ö, Kaya Ö, Rauch C, Nakabo S, Kuehn HS, Swart M, Wang Y, Çelik NI, Molitor A, Ziaee V, Movahedi N, Shahrooei M, Parvaneh N, Alipour-Olyei N, Carapito R, Xu Q, Preite S, Beck DB, Chae JJ, Nehrebecky M, Ombrello AK, Hoffmann P, Romeo T, Deuitch NT, Matthíasardóttir B, Mullikin J, Komarow H, Stoddard J, Niemela J, Dobbs K, Sweeney CL, Anderton H, Lawlor KE, Yoshitomi H, Yang D, Boehm M, Davis J, Mudd P, Randazzo D, Tsai WL, Gadina M, Kaplan MJ, Toguchida J, Mayer CT, Rosenzweig SD, Notarangelo LD, Iwai K, Silke J, Schwartzberg PL, Boisson B, Casanova JL, Bahram S, Rao AP, Peltzer N, Walczak H, Lalaoui N, Aksentijevich I, Kastner DL. Oda H, et al. Among authors: sweeney cl. Nat Immunol. 2024 May;25(5):764-777. doi: 10.1038/s41590-024-01817-w. Epub 2024 Apr 12. Nat Immunol. 2024. PMID: 38609546 Free PMC article.
Lentivector cryptic splicing mediates increase in CD34+ clones expressing truncated HMGA2 in human X-linked severe combined immunodeficiency.
De Ravin SS, Liu S, Sweeney CL, Brault J, Whiting-Theobald N, Ma M, Liu T, Choi U, Lee J, O'Brien SA, Quackenbush P, Estwick T, Karra A, Docking E, Kwatemaa N, Guo S, Su L, Sun Z, Zhou S, Puck J, Cowan MJ, Notarangelo LD, Kang E, Malech HL, Wu X. De Ravin SS, et al. Among authors: sweeney cl. Nat Commun. 2022 Jun 28;13(1):3710. doi: 10.1038/s41467-022-31344-x. Nat Commun. 2022. PMID: 35764638 Free PMC article. Clinical Trial.
CRISPR-targeted MAGT1 insertion restores XMEN patient hematopoietic stem cells and lymphocytes.
Brault J, Liu T, Bello E, Liu S, Sweeney CL, Meis RJ, Koontz S, Corsino C, Choi U, Vayssiere G, Bosticardo M, Dowdell K, Lazzarotto CR, Clark AB, Notarangelo LD, Ravell JC, Lenardo MJ, Kleinstiver BP, Tsai SQ, Wu X, Dahl GA, Malech HL, De Ravin SS. Brault J, et al. Among authors: sweeney cl. Blood. 2021 Dec 30;138(26):2768-2780. doi: 10.1182/blood.2021011192. Blood. 2021. PMID: 34086870 Free PMC article.
Rhesus iPSC Safe Harbor Gene-Editing Platform for Stable Expression of Transgenes in Differentiated Cells of All Germ Layers.
Hong SG, Yada RC, Choi K, Carpentier A, Liang TJ, Merling RK, Sweeney CL, Malech HL, Jung M, Corat MAF, AlJanahi AA, Lin Y, Liu H, Tunc I, Wang X, Palisoc M, Pittaluga S, Boehm M, Winkler T, Zou J, Dunbar CE. Hong SG, et al. Among authors: sweeney cl. Mol Ther. 2017 Jan 4;25(1):44-53. doi: 10.1016/j.ymthe.2016.10.007. Epub 2017 Jan 4. Mol Ther. 2017. PMID: 28129126 Free PMC article.
Differentiation of human and murine induced pluripotent stem cells to microglia-like cells.
Pandya H, Shen MJ, Ichikawa DM, Sedlock AB, Choi Y, Johnson KR, Kim G, Brown MA, Elkahloun AG, Maric D, Sweeney CL, Gossa S, Malech HL, McGavern DB, Park JK. Pandya H, et al. Among authors: sweeney cl. Nat Neurosci. 2017 May;20(5):753-759. doi: 10.1038/nn.4534. Epub 2017 Mar 2. Nat Neurosci. 2017. PMID: 28253233 Free PMC article.
Targeted gene addition in human CD34(+) hematopoietic cells for correction of X-linked chronic granulomatous disease.
De Ravin SS, Reik A, Liu PQ, Li L, Wu X, Su L, Raley C, Theobald N, Choi U, Song AH, Chan A, Pearl JR, Paschon DE, Lee J, Newcombe H, Koontz S, Sweeney C, Shivak DA, Zarember KA, Peshwa MV, Gregory PD, Urnov FD, Malech HL. De Ravin SS, et al. Nat Biotechnol. 2016 Apr;34(4):424-9. doi: 10.1038/nbt.3513. Epub 2016 Mar 7. Nat Biotechnol. 2016. PMID: 26950749 Free PMC article.
The promise of in vivo HSC prime editing.
Sweeney CL, De Ravin SS. Sweeney CL, et al. Blood. 2023 Apr 27;141(17):2039-2040. doi: 10.1182/blood.2023019922. Blood. 2023. PMID: 37103948 Free article. No abstract available.
Functional neutrophils from human ES cells.
Sweeney CL, Malech HL. Sweeney CL, et al. Blood. 2009 Jun 25;113(26):6503-5. doi: 10.1182/blood-2009-04-214320. Blood. 2009. PMID: 19556431 Free PMC article.
Gene Editing in Chronic Granulomatous Disease.
Sweeney CL, Merling RK, De Ravin SS, Choi U, Malech HL. Sweeney CL, et al. Methods Mol Biol. 2019;1982:623-665. doi: 10.1007/978-1-4939-9424-3_36. Methods Mol Biol. 2019. PMID: 31172498
45 results