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A novel therapeutic strategy for skeletal disorders: Proof of concept of gene therapy for X-linked hypophosphatemia.
Zhukouskaya VV, Jauze L, Charles S, Leborgne C, Hilliquin S, Sadoine J, Slimani L, Baroukh B, van Wittenberghe L, Danièle N, Rajas F, Linglart A, Mingozzi F, Chaussain C, Bardet C, Ronzitti G. Zhukouskaya VV, et al. Among authors: daniele n. Sci Adv. 2021 Oct 29;7(44):eabj5018. doi: 10.1126/sciadv.abj5018. Epub 2021 Oct 27. Sci Adv. 2021. PMID: 34705504 Free PMC article.
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: daniele n. Nat Commun. 2021 Nov 4;12(1):6393. doi: 10.1038/s41467-021-26744-4. Nat Commun. 2021. PMID: 34737297 Free PMC article.
Gene therapy with secreted acid alpha-glucosidase rescues Pompe disease in a novel mouse model with early-onset spinal cord and respiratory defects.
Colella P, Sellier P, Gomez MJ, Biferi MG, Tanniou G, Guerchet N, Cohen-Tannoudji M, Moya-Nilges M, van Wittenberghe L, Daniele N, Gjata B, Krijnse-Locker J, Collaud F, Simon-Sola M, Charles S, Cagin U, Mingozzi F. Colella P, et al. Among authors: daniele n. EBioMedicine. 2020 Nov;61:103052. doi: 10.1016/j.ebiom.2020.103052. Epub 2020 Oct 9. EBioMedicine. 2020. PMID: 33039711 Free PMC article.
Synergism of dual AAV gene therapy and rapamycin rescues GSDIII phenotype in muscle and liver.
Jauze L, Vie M, Miagoux Q, Rossiaud L, Vidal P, Montalvo-Romeral V, Saliba H, Jarrige M, Polveche H, Nozi J, Le Brun PR, Bocchialini L, Francois A, Cosette J, Rouillon J, Collaud F, Bordier F, Bertil-Froidevaux E, Georger C, van Wittenberghe L, Miranda A, Daniele NF, Gross DA, Hoch L, Nissan X, Ronzitti G. Jauze L, et al. Among authors: daniele nf. JCI Insight. 2024 May 16;9(11):e172614. doi: 10.1172/jci.insight.172614. JCI Insight. 2024. PMID: 38753465 Free PMC article.
A functional mini-GDE transgene corrects impairment in models of glycogen storage disease type III.
Gardin A, Rouillon J, Montalvo-Romeral V, Rossiaud L, Vidal P, Launay R, Vie M, Krimi Benchekroun Y, Cosette J, Bertin B, La Bella T, Dubreuil G, Nozi J, Jauze L, Fragnoud R, Daniele N, Van Wittenberghe L, Esque J, André I, Nissan X, Hoch L, Ronzitti G. Gardin A, et al. Among authors: daniele n. J Clin Invest. 2024 Jan 16;134(2):e172018. doi: 10.1172/JCI172018. J Clin Invest. 2024. PMID: 38015640 Free PMC article.
Linking Migraine to Gut Dysbiosis and Chronic Non-Communicable Diseases.
Di Lauro M, Guerriero C, Cornali K, Albanese M, Costacurta M, Mercuri NB, Di Daniele N, Noce A. Di Lauro M, et al. Among authors: di daniele n. Nutrients. 2023 Oct 11;15(20):4327. doi: 10.3390/nu15204327. Nutrients. 2023. PMID: 37892403 Free PMC article. Review.
267 results