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Page 1
Characterization of a recombinant adeno-associated virus type 2 Reference Standard Material.
Lock M, McGorray S, Auricchio A, Ayuso E, Beecham EJ, Blouin-Tavel V, Bosch F, Bose M, Byrne BJ, Caton T, Chiorini JA, Chtarto A, Clark KR, Conlon T, Darmon C, Doria M, Douar A, Flotte TR, Francis JD, Francois A, Giacca M, Korn MT, Korytov I, Leon X, Leuchs B, Lux G, Melas C, Mizukami H, Moullier P, Müller M, Ozawa K, Philipsberg T, Poulard K, Raupp C, Rivière C, Roosendaal SD, Samulski RJ, Soltys SM, Surosky R, Tenenbaum L, Thomas DL, van Montfort B, Veres G, Wright JF, Xu Y, Zelenaia O, Zentilin L, Snyder RO. Lock M, et al. Among authors: leon x. Hum Gene Ther. 2010 Oct;21(10):1273-85. doi: 10.1089/hum.2009.223. Hum Gene Ther. 2010. PMID: 20486768 Free PMC article.
Manufacturing and characterization of a recombinant adeno-associated virus type 8 reference standard material.
Ayuso E, Blouin V, Lock M, McGorray S, Leon X, Alvira MR, Auricchio A, Bucher S, Chtarto A, Clark KR, Darmon C, Doria M, Fountain W, Gao G, Gao K, Giacca M, Kleinschmidt J, Leuchs B, Melas C, Mizukami H, Müller M, Noordman Y, Bockstael O, Ozawa K, Pythoud C, Sumaroka M, Surosky R, Tenenbaum L, van der Linden I, Weins B, Wright JF, Zhang X, Zentilin L, Bosch F, Snyder RO, Moullier P. Ayuso E, et al. Among authors: leon x. Hum Gene Ther. 2014 Nov;25(11):977-87. doi: 10.1089/hum.2014.057. Hum Gene Ther. 2014. PMID: 25275822 Free PMC article.
Biochemical, histological and functional correction of mucopolysaccharidosis type IIIB by intra-cerebrospinal fluid gene therapy.
Ribera A, Haurigot V, Garcia M, Marcó S, Motas S, Villacampa P, Maggioni L, León X, Molas M, Sánchez V, Muñoz S, Leborgne C, Moll X, Pumarola M, Mingozzi F, Ruberte J, Añor S, Bosch F. Ribera A, et al. Among authors: leon x. Hum Mol Genet. 2015 Apr 1;24(7):2078-95. doi: 10.1093/hmg/ddu727. Epub 2014 Dec 18. Hum Mol Genet. 2015. PMID: 25524704
FGF21 gene therapy as treatment for obesity and insulin resistance.
Jimenez V, Jambrina C, Casana E, Sacristan V, Muñoz S, Darriba S, Rodó J, Mallol C, Garcia M, León X, Marcó S, Ribera A, Elias I, Casellas A, Grass I, Elias G, Ferré T, Motas S, Franckhauser S, Mulero F, Navarro M, Haurigot V, Ruberte J, Bosch F. Jimenez V, et al. Among authors: leon x. EMBO Mol Med. 2018 Aug;10(8):e8791. doi: 10.15252/emmm.201708791. EMBO Mol Med. 2018. PMID: 29987000 Free PMC article.
Treatment of skeletal and non-skeletal alterations of Mucopolysaccharidosis type IVA by AAV-mediated gene therapy.
Bertolin J, Sánchez V, Ribera A, Jaén ML, Garcia M, Pujol A, Sánchez X, Muñoz S, Marcó S, Pérez J, Elias G, León X, Roca C, Jimenez V, Otaegui P, Mulero F, Navarro M, Ruberte J, Bosch F. Bertolin J, et al. Among authors: leon x. Nat Commun. 2021 Sep 9;12(1):5343. doi: 10.1038/s41467-021-25697-y. Nat Commun. 2021. PMID: 34504088 Free PMC article.
Seven-year follow-up of durability and safety of AAV CNS gene therapy for a lysosomal storage disorder in a large animal.
Marcó S, Haurigot V, Jaén ML, Ribera A, Sánchez V, Molas M, Garcia M, León X, Roca C, Sánchez X, Bertolin J, Pérez J, Elias G, Navarro M, Carretero A, Pumarola M, Andaluz A, Espada Y, Añor S, Bosch F. Marcó S, et al. Among authors: leon x. Mol Ther Methods Clin Dev. 2021 Oct 5;23:370-389. doi: 10.1016/j.omtm.2021.09.017. eCollection 2021 Dec 10. Mol Ther Methods Clin Dev. 2021. PMID: 34761052 Free PMC article.
214 results