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Year Number of Results
2002 1
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28 results

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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
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.
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
High-fidelity PAMless base editing of hematopoietic stem cells to treat chronic granulomatous disease.
Bzhilyanskaya V, Ma L, Liu S, Fox LR, Whittaker MN, Meis RJ, Choi U, Lawson A, Ma M, Theobald N, Burkett S, Sweeney CL, Lazzarotto CR, Tsai SQ, Lack JB, Wu X, Dahl GA, Malech HL, Kleinstiver BP, De Ravin SS. Bzhilyanskaya V, et al. Among authors: sweeney cl. Sci Transl Med. 2024 Oct 16;16(769):eadj6779. doi: 10.1126/scitranslmed.adj6779. Epub 2024 Oct 16. Sci Transl Med. 2024. PMID: 39413163
Self-organized yolk sac-like organoids allow for scalable generation of multipotent hematopoietic progenitor cells from induced pluripotent stem cells.
Tamaoki N, Siebert S, Maeda T, Ha NH, Good ML, Huang Y, Vodnala SK, Haro-Mora JJ, Uchida N, Tisdale JF, Sweeney CL, Choi U, Brault J, Koontz S, Malech HL, Yamazaki Y, Isonaka R, Goldstein DS, Kimura M, Takebe T, Zou J, Stroncek DF, Robey PG, Kruhlak MJ, Restifo NP, Vizcardo R. Tamaoki N, et al. Among authors: sweeney cl. Cell Rep Methods. 2023 Apr 24;3(4):100460. doi: 10.1016/j.crmeth.2023.100460. eCollection 2023 Apr 24. Cell Rep Methods. 2023. PMID: 37159663 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.
Enhanced homology-directed repair for highly efficient gene editing in hematopoietic stem/progenitor cells.
De Ravin SS, Brault J, Meis RJ, Liu S, Li L, Pavel-Dinu M, Lazzarotto CR, Liu T, Koontz SM, Choi U, Sweeney CL, Theobald N, Lee G, Clark AB, Burkett SS, Kleinstiver BP, Porteus MH, Tsai S, Kuhns DB, Dahl GA, Headey S, Wu X, Malech HL. De Ravin SS, et al. Among authors: sweeney cl. Blood. 2021 May 13;137(19):2598-2608. doi: 10.1182/blood.2020008503. Blood. 2021. PMID: 33623984 Free PMC article.
Gene-edited pseudogene resurrection corrects p47phox-deficient chronic granulomatous disease.
Merling RK, Kuhns DB, Sweeney CL, Wu X, Burkett S, Chu J, Lee J, Koontz S, Di Pasquale G, Afione SA, Chiorini JA, Kang EM, Choi U, De Ravin SS, Malech HL. Merling RK, et al. Among authors: sweeney cl. Blood Adv. 2016 Dec 28;1(4):270-278. doi: 10.1182/bloodadvances.2016001214. eCollection 2017 Jan 10. Blood Adv. 2016. PMID: 29296942 Free PMC article.
Homozygous IL37 mutation associated with infantile inflammatory bowel disease.
Zhang ZZ, Zhang Y, He T, Sweeney CL, Baris S, Karakoc-Aydiner E, Yao Y, Ertem D, Matthews HF, Gonzaga-Jauregui C, Malech HL, Su HC, Ozen A, Smith KGC, Lenardo MJ. Zhang ZZ, et al. Among authors: sweeney cl. Proc Natl Acad Sci U S A. 2021 Mar 9;118(10):e2009217118. doi: 10.1073/pnas.2009217118. Proc Natl Acad Sci U S A. 2021. PMID: 33674380 Free PMC article. Clinical Trial.
28 results