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Lentiviral gene therapy for X-linked chronic granulomatous disease.
Kohn DB, Booth C, Kang EM, Pai SY, Shaw KL, Santilli G, Armant M, Buckland KF, Choi U, De Ravin SS, Dorsey MJ, Kuo CY, Leon-Rico D, Rivat C, Izotova N, Gilmour K, Snell K, Dip JX, Darwish J, Morris EC, Terrazas D, Wang LD, Bauser CA, Paprotka T, Kuhns DB, Gregg J, Raymond HE, Everett JK, Honnet G, Biasco L, Newburger PE, Bushman FD, Grez M, Gaspar HB, Williams DA, Malech HL, Galy A, Thrasher AJ; Net4CGD consortium. Kohn DB, et al. Among authors: thrasher aj. Nat Med. 2020 Feb;26(2):200-206. doi: 10.1038/s41591-019-0735-5. Epub 2020 Jan 27. Nat Med. 2020. PMID: 31988463 Free PMC article. Clinical Trial.
Severe hematopoietic stem cell inflammation compromises chronic granulomatous disease gene therapy.
Sobrino S, Magnani A, Semeraro M, Martignetti L, Cortal A, Denis A, Couzin C, Picard C, Bustamante J, Magrin E, Joseph L, Roudaut C, Gabrion A, Soheili T, Cordier C, Lortholary O, Lefrere F, Rieux-Laucat F, Casanova JL, Bodard S, Boddaert N, Thrasher AJ, Touzot F, Taque S, Suarez F, Marcais A, Guilloux A, Lagresle-Peyrou C, Galy A, Rausell A, Blanche S, Cavazzana M, Six E. Sobrino S, et al. Among authors: thrasher aj. Cell Rep Med. 2023 Feb 21;4(2):100919. doi: 10.1016/j.xcrm.2023.100919. Epub 2023 Jan 26. Cell Rep Med. 2023. PMID: 36706754 Free PMC article. Clinical Trial.
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.
Assessment of Pre-Clinical Liver Models Based on Their Ability to Predict the Liver-Tropism of Adeno-Associated Virus Vectors.
Westhaus A, Cabanes-Creus M, Dilworth KL, Zhu E, Salas Gómez D, Navarro RG, Amaya AK, Scott S, Kwiatek M, McCorkindale AL, Hayman TE, Frahm S, Perocheau DP, Tran BM, Vincan E, Wong SL, Waters SA, Riddiough GE, Perini MV, Wilson LOW, Baruteau J, Diecke S, González-Aseguinolaza G, Santilli G, Thrasher AJ, Alexander IE, Lisowski L. Westhaus A, et al. Among authors: thrasher aj. Hum Gene Ther. 2023 Apr;34(7-8):273-288. doi: 10.1089/hum.2022.188. Hum Gene Ther. 2023. PMID: 36927149 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.
413 results