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23 results

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Page 1
ARI0003: Co-transduced CD19/BCMA dual-targeting CAR-T cells for the treatment of non-Hodgkin lymphoma.
Bachiller M, Barceló-Genestar N, Rodriguez-Garcia A, Alserawan L, Dobaño-López C, Giménez-Alejandre M, Castellsagué J, Colell S, Otero-Mateo M, Antoñana-Vildosola A, Español-Rego M, Ferruz N, Pascal M, Martín-Antonio B, Anguela XM, Fillat C, Olesti E, Calvo G, Juan M, Delgado J, Pérez-Galán P, Urbano-Ispizua Á, Guedan S. Bachiller M, et al. Among authors: anguela xm. Mol Ther. 2024 Nov 19:S1525-0016(24)00755-X. doi: 10.1016/j.ymthe.2024.11.028. Online ahead of print. Mol Ther. 2024. PMID: 39563035 Free article.
Preclinical assessment of an optimized AAV-FVIII vector in mice and non-human primates for the treatment of hemophilia A.
Elkouby L, Armour SM, Toso R, DiPietro M, Davidson RJ, Nguyen GN, Willet M, Kutza S, Silverberg J, Frick J, Crosariol M, Wang Y, Wang C, High KA, Sabatino DE, Anguela XM. Elkouby L, et al. Among authors: anguela xm. Mol Ther Methods Clin Dev. 2021 Nov 24;24:20-29. doi: 10.1016/j.omtm.2021.11.005. eCollection 2022 Mar 10. Mol Ther Methods Clin Dev. 2021. PMID: 34977269 Free PMC article.
Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A.
George LA, Monahan PE, Eyster ME, Sullivan SK, Ragni MV, Croteau SE, Rasko JEJ, Recht M, Samelson-Jones BJ, MacDougall A, Jaworski K, Noble R, Curran M, Kuranda K, Mingozzi F, Chang T, Reape KZ, Anguela XM, High KA. George LA, et al. Among authors: anguela xm. N Engl J Med. 2021 Nov 18;385(21):1961-1973. doi: 10.1056/NEJMoa2104205. N Engl J Med. 2021. PMID: 34788507 Free PMC article. Clinical Trial.
Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates.
Costa-Verdera H, Collaud F, Riling CR, Sellier P, Nordin JML, Preston GM, Cagin U, Fabregue J, Barral S, Moya-Nilges M, Krijnse-Locker J, van Wittenberghe L, Daniele N, Gjata B, Cosette J, Abad C, Simon-Sola M, Charles S, Li M, Crosariol M, Antrilli T, Quinn WJ 3rd, Gross DA, Boyer O, Anguela XM, Armour SM, Colella P, Ronzitti G, Mingozzi F. Costa-Verdera H, et al. Among authors: anguela xm. Nat Commun. 2021 Nov 4;12(1):6393. doi: 10.1038/s41467-021-26744-4. Nat Commun. 2021. PMID: 34737297 Free PMC article.
Experimental Variables that Affect Human Hepatocyte AAV Transduction in Liver Chimeric Mice.
Zou C, Vercauteren KOA, Michailidis E, Kabbani M, Zoluthkin I, Quirk C, Chiriboga L, Yazicioglu M, Anguela XM, Meuleman P, High KA, Herzog RW, de Jong YP. Zou C, et al. Among authors: anguela xm. Mol Ther Methods Clin Dev. 2020 Jun 2;18:189-198. doi: 10.1016/j.omtm.2020.05.033. eCollection 2020 Sep 11. Mol Ther Methods Clin Dev. 2020. PMID: 32637450 Free PMC article.
Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B.
George LA, Ragni MV, Rasko JEJ, Raffini LJ, Samelson-Jones BJ, Ozelo M, Hazbon M, Runowski AR, Wellman JA, Wachtel K, Chen Y, Anguela XM, Kuranda K, Mingozzi F, High KA. George LA, et al. Among authors: anguela xm. Mol Ther. 2020 Sep 2;28(9):2073-2082. doi: 10.1016/j.ymthe.2020.06.001. Epub 2020 Jun 10. Mol Ther. 2020. PMID: 32559433 Free PMC article. Clinical Trial.
IgG-cleaving endopeptidase enables in vivo gene therapy in the presence of anti-AAV neutralizing antibodies.
Leborgne C, Barbon E, Alexander JM, Hanby H, Delignat S, Cohen DM, Collaud F, Muraleetharan S, Lupo D, Silverberg J, Huang K, van Wittengerghe L, Marolleau B, Miranda A, Fabiano A, Daventure V, Beck H, Anguela XM, Ronzitti G, Armour SM, Lacroix-Desmazes S, Mingozzi F. Leborgne C, et al. Among authors: anguela xm. Nat Med. 2020 Jul;26(7):1096-1101. doi: 10.1038/s41591-020-0911-7. Epub 2020 Jun 1. Nat Med. 2020. PMID: 32483358 Free article.
Entering the Modern Era of Gene Therapy.
Anguela XM, High KA. Anguela XM, et al. Annu Rev Med. 2019 Jan 27;70:273-288. doi: 10.1146/annurev-med-012017-043332. Epub 2018 Nov 26. Annu Rev Med. 2019. PMID: 30477394 Review.
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