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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: perez j. 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.
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: perez j. Nat Commun. 2021 Sep 9;12(1):5343. doi: 10.1038/s41467-021-25697-y. Nat Commun. 2021. PMID: 34504088 Free PMC article.
Early Onset Active Inflammatory Bowel Disease Is Associated With Psychiatric Comorbidities: A Multi-Network Propensity-Matched Cohort Study.
Nadeem A, Donohue S, Shah FZ, Perez JA, Harper E, Sinh P, Padival R, Cooper G, Katz J, Cominelli F, Regueiro M, Mansoor E. Nadeem A, et al. Among authors: perez ja. Crohns Colitis 360. 2024 Dec 20;7(1):otae066. doi: 10.1093/crocol/otae066. eCollection 2025 Jan. Crohns Colitis 360. 2024. PMID: 39749210 Free PMC article.
5,338 results