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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: jaen ml. Nat Commun. 2021 Sep 9;12(1):5343. doi: 10.1038/s41467-021-25697-y. Nat Commun. 2021. PMID: 34504088 Free PMC article.
Treatment of infantile-onset Pompe disease in a rat model with muscle-directed AAV gene therapy.
Muñoz S, Bertolin J, Jimenez V, Jaén ML, Garcia M, Pujol A, Vilà L, Sacristan V, Barbon E, Ronzitti G, El Andari J, Tulalamba W, Pham QH, Ruberte J, VandenDriessche T, Chuah MK, Grimm D, Mingozzi F, Bosch F. Muñoz S, et al. Among authors: jaen ml. Mol Metab. 2024 Mar;81:101899. doi: 10.1016/j.molmet.2024.101899. Epub 2024 Feb 10. Mol Metab. 2024. PMID: 38346589 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: jaen ml. 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.
Long-Term Efficacy and Safety of Insulin and Glucokinase Gene Therapy for Diabetes: 8-Year Follow-Up in Dogs.
Jaén ML, Vilà L, Elias I, Jimenez V, Rodó J, Maggioni L, Ruiz-de Gopegui R, Garcia M, Muñoz S, Callejas D, Ayuso E, Ferré T, Grifoll I, Andaluz A, Ruberte J, Haurigot V, Bosch F. Jaén ML, et al. Mol Ther Methods Clin Dev. 2017 Apr 5;6:1-7. doi: 10.1016/j.omtm.2017.03.008. eCollection 2017 Sep 15. Mol Ther Methods Clin Dev. 2017. PMID: 28626777 Free PMC article.