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3,858 results

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Attenuation of Heparan Sulfate Proteoglycan Binding Enhances In Vivo Transduction of Human Primary Hepatocytes with AAV2.
Cabanes-Creus M, Westhaus A, Navarro RG, Baltazar G, Zhu E, Amaya AK, Liao SHY, Scott S, Sallard E, Dilworth KL, Rybicki A, Drouyer M, Hallwirth CV, Bennett A, Santilli G, Thrasher AJ, Agbandje-McKenna M, Alexander IE, Lisowski L. Cabanes-Creus M, et al. Among authors: scott s. Mol Ther Methods Clin Dev. 2020 May 13;17:1139-1154. doi: 10.1016/j.omtm.2020.05.004. eCollection 2020 Jun 12. Mol Ther Methods Clin Dev. 2020. PMID: 32490035 Free PMC article.
Single amino acid insertion allows functional transduction of murine hepatocytes with human liver tropic AAV capsids.
Cabanes-Creus M, Navarro RG, Liao SHY, Baltazar G, Drouyer M, Zhu E, Scott S, Luong C, Wilson LOW, Alexander IE, Lisowski L. Cabanes-Creus M, et al. Among authors: scott s. Mol Ther Methods Clin Dev. 2021 Apr 24;21:607-620. doi: 10.1016/j.omtm.2021.04.010. eCollection 2021 Jun 11. Mol Ther Methods Clin Dev. 2021. PMID: 34095344 Free PMC article.
Novel human liver-tropic AAV variants define transferable domains that markedly enhance the human tropism of AAV7 and AAV8.
Cabanes-Creus M, Navarro RG, Zhu E, Baltazar G, Liao SHY, Drouyer M, Amaya AK, Scott S, Nguyen LH, Westhaus A, Hebben M, Wilson LOW, Thrasher AJ, Alexander IE, Lisowski L. Cabanes-Creus M, et al. Among authors: scott s. Mol Ther Methods Clin Dev. 2021 Nov 25;24:88-101. doi: 10.1016/j.omtm.2021.11.011. eCollection 2022 Mar 10. Mol Ther Methods Clin Dev. 2021. PMID: 34977275 Free PMC article.
Characterization of the humanized FRG mouse model and development of an AAV-LK03 variant with improved liver lobular biodistribution.
Cabanes-Creus M, Navarro RG, Liao SHY, Scott S, Carlessi R, Roca-Pinilla R, Knight M, Baltazar G, Zhu E, Jones M, Denisenko E, Forrest ARR, Alexander IE, Tirnitz-Parker JEE, Lisowski L. Cabanes-Creus M, et al. Among authors: scott s. Mol Ther Methods Clin Dev. 2023 Jan 2;28:220-237. doi: 10.1016/j.omtm.2022.12.014. eCollection 2023 Mar 9. Mol Ther Methods Clin Dev. 2023. PMID: 36700121 Free PMC article.
Development of new adeno-associated virus capsid variants for targeted gene delivery to human cardiomyocytes.
Kok CY, Tsurusaki S, Cabanes-Creus M, Igoor S, Rao R, Skelton R, Liao SHY, Ginn SL, Knight M, Scott S, Mietzsch M, Fitzsimmons R, Miller J, Mohamed TMA, McKenna R, Chong JJH, Hill AP, Hudson JE, Alexander IE, Lisowski L, Kizana E. Kok CY, et al. Among authors: scott s. Mol Ther Methods Clin Dev. 2023 Aug 18;30:459-473. doi: 10.1016/j.omtm.2023.08.010. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37674904 Free PMC article.
Novel AAV variants with improved tropism for human Schwann cells.
Drouyer M, Chu TH, Labit E, Haase F, Navarro RG, Nazareth D, Rosin N, Merjane J, Scott S, Cabanes-Creus M, Westhaus A, Zhu E, Midha R, Alexander IE, Biernaskie J, Ginn SL, Lisowski L. Drouyer M, et al. Among authors: scott s. Mol Ther Methods Clin Dev. 2024 Mar 11;32(2):101234. doi: 10.1016/j.omtm.2024.101234. eCollection 2024 Jun 13. Mol Ther Methods Clin Dev. 2024. PMID: 38558569 Free PMC article.
Restoring the natural tropism of AAV2 vectors for human liver.
Cabanes-Creus M, Hallwirth CV, Westhaus A, Ng BH, Liao SHY, Zhu E, Navarro RG, Baltazar G, Drouyer M, Scott S, Logan GJ, Santilli G, Bennett A, Ginn SL, McCaughan G, Thrasher AJ, Agbandje-McKenna M, Alexander IE, Lisowski L. Cabanes-Creus M, et al. Among authors: scott s. Sci Transl Med. 2020 Sep 9;12(560):eaba3312. doi: 10.1126/scitranslmed.aba3312. Sci Transl Med. 2020. PMID: 32908003
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: scott s. 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.
3,858 results