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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.
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 Jun 5;32(6):1672-1686. doi: 10.1016/j.ymthe.2024.03.032. Epub 2024 Mar 27. Mol Ther. 2024. PMID: 38549377
Replication competent retrovirus testing (RCR) in the National Gene Vector Biorepository: No evidence of RCR in 1,595 post-treatment peripheral blood samples obtained from 60 clinical trials.
Cornetta K, Yao J, House K, Duffy L, Adusumilli PS, Beyer R, Booth C, Brenner M, Curran K, Grilley B, Heslop H, Hinrichs CS, Kaplan RN, Kiem HP, Kochenderfer J, Kohn DB, Mailankody S, Norberg SM, O'Cearbhaill RE, Pappas J, Park J, Ramos C, Ribas A, Rivière I, Rosenberg SA, Sauter C, Shah NN, Slovin SF, Thrasher A, Williams DA, Lin TY. Cornetta K, et al. Mol Ther. 2023 Mar 1;31(3):801-809. doi: 10.1016/j.ymthe.2022.12.006. Epub 2022 Dec 14. Mol Ther. 2023. PMID: 36518078 Free PMC article.
Comprehensive Cancer-Predisposition Gene Testing in an Adult Multiple Primary Tumor Series Shows a Broad Range of Deleterious Variants and Atypical Tumor Phenotypes.
Whitworth J, Smith PS, Martin JE, West H, Luchetti A, Rodger F, Clark G, Carss K, Stephens J, Stirrups K, Penkett C, Mapeta R, Ashford S, Megy K, Shakeel H, Ahmed M, Adlard J, Barwell J, Brewer C, Casey RT, Armstrong R, Cole T, Evans DG, Fostira F, Greenhalgh L, Hanson H, Henderson A, Hoffman J, Izatt L, Kumar A, Kwong A, Lalloo F, Ong KR, Paterson J, Park SM, Chen-Shtoyerman R, Searle C, Side L, Skytte AB, Snape K, Woodward ER; NIHR BioResource Rare Diseases Consortium; Tischkowitz MD, Maher ER. Whitworth J, et al. Am J Hum Genet. 2018 Jul 5;103(1):3-18. doi: 10.1016/j.ajhg.2018.04.013. Epub 2018 Jun 14. Am J Hum Genet. 2018. PMID: 29909963 Free PMC article.
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.
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