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1954 2
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2000 7
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2002 12
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2004 18
2005 16
2006 12
2007 27
2008 21
2009 18
2010 17
2011 20
2012 28
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Page 1
Showing results for george l
Search for Geoge L instead (1 results)
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease.
Musunuru K, Grandinette SA, Wang X, Hudson TR, Briseno K, Berry AM, Hacker JL, Hsu A, Silverstein RA, Hille LT, Ogul AN, Robinson-Garvin NA, Small JC, McCague S, Burke SM, Wright CM, Bick S, Indurthi V, Sharma S, Jepperson M, Vakulskas CA, Collingwood M, Keogh K, Jacobi A, Sturgeon M, Brommel C, Schmaljohn E, Kurgan G, Osborne T, Zhang H, Kinney K, Rettig G, Barbosa CJ, Semple SC, Tam YK, Lutz C, George LA, Kleinstiver BP, Liu DR, Ng K, Kassim SH, Giannikopoulos P, Alameh MG, Urnov FD, Ahrens-Nicklas RC. Musunuru K, et al. Among authors: george la. N Engl J Med. 2025 Jun 12;392(22):2235-2243. doi: 10.1056/NEJMoa2504747. Epub 2025 May 15. N Engl J Med. 2025. PMID: 40373211
Current Clinical Applications of In Vivo Gene Therapy with AAVs.
Mendell JR, Al-Zaidy SA, Rodino-Klapac LR, Goodspeed K, Gray SJ, Kay CN, Boye SL, Boye SE, George LA, Salabarria S, Corti M, Byrne BJ, Tremblay JP. Mendell JR, et al. Among authors: george la. Mol Ther. 2021 Feb 3;29(2):464-488. doi: 10.1016/j.ymthe.2020.12.007. Epub 2020 Dec 10. Mol Ther. 2021. PMID: 33309881 Free PMC article. Review.
Adeno-Associated Virus Gene Therapy for Hemophilia.
Samelson-Jones BJ, George LA. Samelson-Jones BJ, et al. Among authors: george la. Annu Rev Med. 2023 Jan 27;74:231-247. doi: 10.1146/annurev-med-043021-033013. Epub 2022 Sep 14. Annu Rev Med. 2023. PMID: 36103998 Free PMC article. Review.
Roctavian gene therapy for hemophilia A.
Samelson-Jones BJ, Small JC, George LA. Samelson-Jones BJ, et al. Among authors: george la. Blood Adv. 2024 Oct 8;8(19):5179-5189. doi: 10.1182/bloodadvances.2023011847. Blood Adv. 2024. PMID: 38991118 Free PMC article. Review.
Intra-condensate demixing of TDP-43 inside stress granules generates pathological aggregates.
Yan X, Kuster D, Mohanty P, Nijssen J, Pombo-García K, Rizuan A, Franzmann TM, Sergeeva A, Passos PM, George L, Wang SH, Shenoy J, Danielson HL, Honigmann A, Ayala YM, Fawzi NL, Mittal J, Alberti S, Hyman AA. Yan X, et al. Among authors: george l. bioRxiv [Preprint]. 2024 May 24:2024.01.23.576837. doi: 10.1101/2024.01.23.576837. bioRxiv. 2024. Update in: Cell. 2025 Jul 24;188(15):4123-4140.e18. doi: 10.1016/j.cell.2025.04.039. PMID: 38328053 Free PMC article. Updated. Preprint.
Intra-condensate demixing of TDP-43 inside stress granules generates pathological aggregates.
Yan X, Kuster D, Mohanty P, Nijssen J, Pombo-García K, Garcia Morato J, Rizuan A, Franzmann TM, Sergeeva A, Ly AM, Liu F, Passos PM, George L, Wang SH, Shenoy J, Danielson HL, Ozguney B, Honigmann A, Ayala YM, Fawzi NL, Dickson DW, Rossoll W, Mittal J, Alberti S, Hyman AA. Yan X, et al. Among authors: george l. Cell. 2025 Jul 24;188(15):4123-4140.e18. doi: 10.1016/j.cell.2025.04.039. Epub 2025 May 23. Cell. 2025. PMID: 40412392 Free article.
Current clinical applications of AAV-mediated gene therapy.
Byrne BJ, Flanigan KM, Matesanz SE, Finkel RS, Waldrop MA, D'Ambrosio ES, Johnson NE, Smith BK, Bönnemann C, Carrig S, Rossano JW, Greenberg B, Lalaguna L, Lara-Pezzi E, Subramony S, Corti M, Mercado-Rodriguez C, Leon-Astudillo C, Ahrens-Nicklas R, Bharucha-Goebel D, Gao G, Gessler DJ, Hwu WL, Chien YH, Lee NC, Boye SL, Boye SE, George LA. Byrne BJ, et al. Among authors: george la. Mol Ther. 2025 Jun 4;33(6):2479-2516. doi: 10.1016/j.ymthe.2025.04.045. Epub 2025 May 5. Mol Ther. 2025. PMID: 40329530 Free article. Review.
Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant.
George LA, Sullivan SK, Giermasz A, Rasko JEJ, Samelson-Jones BJ, Ducore J, Cuker A, Sullivan LM, Majumdar S, Teitel J, McGuinn CE, Ragni MV, Luk AY, Hui D, Wright JF, Chen Y, Liu Y, Wachtel K, Winters A, Tiefenbacher S, Arruda VR, van der Loo JCM, Zelenaia O, Takefman D, Carr ME, Couto LB, Anguela XM, High KA. George LA, et al. N Engl J Med. 2017 Dec 7;377(23):2215-2227. doi: 10.1056/NEJMoa1708538. N Engl J Med. 2017. PMID: 29211678 Free PMC article. Clinical Trial.
Intracellular RNA-tracking methods.
George L, Indig FE, Abdelmohsen K, Gorospe M. George L, et al. Open Biol. 2018 Oct 3;8(10):180104. doi: 10.1098/rsob.180104. Open Biol. 2018. PMID: 30282659 Free PMC article. Review.
1,080 results