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Page 1
Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy.
Haurigot V, Marcó S, Ribera A, Garcia M, Ruzo A, Villacampa P, Ayuso E, Añor S, Andaluz A, Pineda M, García-Fructuoso G, Molas M, Maggioni L, Muñoz S, Motas S, Ruberte J, Mingozzi F, Pumarola M, Bosch F. Haurigot V, et al. Among authors: marco s. J Clin Invest. 2013 Aug 1;123(8):3254-3271. doi: 10.1172/JCI66778. Epub 2013 Jul 1. J Clin Invest. 2013. PMID: 23863627 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: marco s. 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: marco s. 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: marco s. Nat Commun. 2021 Sep 9;12(1):5343. doi: 10.1038/s41467-021-25697-y. Nat Commun. 2021. PMID: 34504088 Free PMC article.
629 results