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Whole-genome sequencing of a sporadic primary immunodeficiency cohort.
Thaventhiran JED, Lango Allen H, Burren OS, Rae W, Greene D, Staples E, Zhang Z, Farmery JHR, Simeoni I, Rivers E, Maimaris J, Penkett CJ, Stephens J, Deevi SVV, Sanchis-Juan A, Gleadall NS, Thomas MJ, Sargur RB, Gordins P, Baxendale HE, Brown M, Tuijnenburg P, Worth A, Hanson S, Linger RJ, Buckland MS, Rayner-Matthews PJ, Gilmour KC, Samarghitean C, Seneviratne SL, Sansom DM, Lynch AG, Megy K, Ellinghaus E, Ellinghaus D, Jorgensen SF, Karlsen TH, Stirrups KE, Cutler AJ, Kumararatne DS, Chandra A, Edgar JDM, Herwadkar A, Cooper N, Grigoriadou S, Huissoon AP, Goddard S, Jolles S, Schuetz C, Boschann F; Primary Immunodeficiency Consortium for the NIHR Bioresource; Lyons PA, Hurles ME, Savic S, Burns SO, Kuijpers TW, Turro E, Ouwehand WH, Thrasher AJ, Smith KGC. Thaventhiran JED, et al. Among authors: thrasher aj. Nature. 2020 Jul;583(7814):90-95. doi: 10.1038/s41586-020-2265-1. Epub 2020 May 6. Nature. 2020. PMID: 32499645 Free PMC article.
Gene silencing dynamics are modulated by transiently active regulatory elements.
Vermunt MW, Luan J, Zhang Z, Thrasher AJ, Huang A, Saari MS, Khandros E, Beagrie RA, Zhang S, Vemulamada P, Brilleman M, Lee K, Yano JA, Giardine BM, Keller CA, Hardison RC, Blobel GA. Vermunt MW, et al. Among authors: thrasher aj. Mol Cell. 2023 Mar 2;83(5):715-730.e6. doi: 10.1016/j.molcel.2023.02.006. Mol Cell. 2023. PMID: 36868189 Free PMC article.
Anti-CD45 PBD-based antibody-drug conjugates are effective targeted conditioning agents for gene therapy and stem cell transplant.
Yeung J, Liao A, Shaw M, Silva S, Vetharoy W, Rico DL, Kirby I, Zammarchi F, Havenith K, de Haan L, van Berkel PH, Sebire N, Ogunbiyi OK, Booth C, Gaspar HB, Thrasher AJ, Chester KA, Amrolia PJ. Yeung J, et al. Among authors: thrasher aj. Mol Ther. 2024 Mar 27:S1525-0016(24)00210-7. doi: 10.1016/j.ymthe.2024.03.032. Online ahead of print. Mol Ther. 2024. PMID: 38549377
Hematopoietic stem cell gene editing rescues B-cell development in X-linked agammaglobulinemia.
Bahal S, Zinicola M, Moula SE, Whittaker TE, Schejtman A, Naseem A, Blanco E, Vetharoy W, Hu YT, Rai R, Gomez-Castaneda E, Cunha-Santos C, Burns SO, Morris EC, Booth C, Turchiano G, Cavazza A, Thrasher AJ, Santilli G. Bahal S, et al. Among authors: thrasher aj. J Allergy Clin Immunol. 2024 Mar 11:S0091-6749(24)00240-9. doi: 10.1016/j.jaci.2024.03.003. Online ahead of print. J Allergy Clin Immunol. 2024. PMID: 38479630 Free article.
Investigating adverse genomic and regulatory changes caused by replacement of the full-length CFTR cDNA using Cas9 and AAV.
Vaidyanathan S, Kerschner JL, Paranjapye A, Sinha V, Lin B, Bedrosian TA, Thrasher AJ, Turchiano G, Harris A, Porteus MH. Vaidyanathan S, et al. Among authors: thrasher aj. Mol Ther Nucleic Acids. 2024 Feb 2;35(1):102134. doi: 10.1016/j.omtn.2024.102134. eCollection 2024 Mar 12. Mol Ther Nucleic Acids. 2024. PMID: 38384445 Free PMC article.
AAV capsid bioengineering in primary human retina models.
Westhaus A, Eamegdool SS, Fernando M, Fuller-Carter P, Brunet AA, Miller AL, Rashwan R, Knight M, Daniszewski M, Lidgerwood GE, Pébay A, Hewitt A, Santilli G, Thrasher AJ, Carvalho LS, Gonzalez-Cordero A, Jamieson RV, Lisowski L. Westhaus A, et al. Among authors: thrasher aj. Sci Rep. 2023 Dec 11;13(1):21946. doi: 10.1038/s41598-023-49112-2. Sci Rep. 2023. PMID: 38081924 Free PMC article.
413 results