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Page 1
Rescue of Advanced Pompe Disease in Mice with Hepatic Expression of Secretable Acid α-Glucosidase.
Cagin U, Puzzo F, Gomez MJ, Moya-Nilges M, Sellier P, Abad C, Van Wittenberghe L, Daniele N, Guerchet N, Gjata B, Collaud F, Charles S, Sola MS, Boyer O, Krijnse-Locker J, Ronzitti G, Colella P, Mingozzi F. Cagin U, et al. Among authors: sola ms. Mol Ther. 2020 Sep 2;28(9):2056-2072. doi: 10.1016/j.ymthe.2020.05.025. Epub 2020 May 30. Mol Ther. 2020. PMID: 32526204 Free PMC article.
A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome.
Ronzitti G, Bortolussi G, van Dijk R, Collaud F, Charles S, Leborgne C, Vidal P, Martin S, Gjata B, Sola MS, van Wittenberghe L, Vignaud A, Veron P, Bosma PJ, Muro AF, Mingozzi F. Ronzitti G, et al. Among authors: sola ms. Mol Ther Methods Clin Dev. 2016 Jul 20;3:16049. doi: 10.1038/mtm.2016.49. eCollection 2016. Mol Ther Methods Clin Dev. 2016. PMID: 27722180 Free PMC article.
Long-term correction of ornithine transcarbamylase deficiency in Spf-Ash mice with a translationally optimized AAV vector.
De Sabbata G, Boisgerault F, Guarnaccia C, Iaconcig A, Bortolussi G, Collaud F, Ronzitti G, Sola MS, Vidal P, Rouillon J, Charles S, Nicastro E, D'Antiga L, Ilyinskii P, Mingozzi F, Kishimoto TK, Muro AF. De Sabbata G, et al. Among authors: sola ms. Mol Ther Methods Clin Dev. 2020 Nov 17;20:169-180. doi: 10.1016/j.omtm.2020.11.005. eCollection 2021 Mar 12. Mol Ther Methods Clin Dev. 2020. PMID: 33473356 Free PMC article.
Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome.
Collaud F, Bortolussi G, Guianvarc'h L, Aronson SJ, Bordet T, Veron P, Charles S, Vidal P, Sola MS, Rundwasser S, Dufour DG, Lacoste F, Luc C, Wittenberghe LV, Martin S, Le Bec C, Bosma PJ, Muro AF, Ronzitti G, Hebben M, Mingozzi F. Collaud F, et al. Among authors: sola ms. Mol Ther Methods Clin Dev. 2018 Dec 31;12:157-174. doi: 10.1016/j.omtm.2018.12.011. eCollection 2019 Mar 15. Mol Ther Methods Clin Dev. 2018. PMID: 30705921 Free PMC article.
Correction: An obesogenic feedforward loop involving PPARγ, acyl-CoA binding protein and GABA receptor.
Anagnostopoulos G, Motiño O, Li S, Carbonnier V, Chen H, Sica V, Durand S, Bourgin M, Aprahamian F, Nirmalathasan N, Donne R, Desdouets C, Sola MS, Kotta K, Montégut L, Lambertucci F, Surdez D, Sandrine G, Delattre O, Maiuri MC, Bravo-San Pedro JM, Martins I, Kroemer G. Anagnostopoulos G, et al. Among authors: sola ms. Cell Death Dis. 2022 May 3;13(5):430. doi: 10.1038/s41419-022-04884-9. Cell Death Dis. 2022. PMID: 35504865 Free PMC article. No abstract available.
An obesogenic feedforward loop involving PPARγ, acyl-CoA binding protein and GABAA receptor.
Anagnostopoulos G, Motiño O, Li S, Carbonnier V, Chen H, Sica V, Durand S, Bourgin M, Aprahamian F, Nirmalathasan N, Donne R, Desdouets C, Sola MS, Kotta K, Montégut L, Lambertucci F, Surdez D, Sandrine G, Delattre O, Maiuri MC, Bravo-San Pedro JM, Martins I, Kroemer G. Anagnostopoulos G, et al. Among authors: sola ms. Cell Death Dis. 2022 Apr 18;13(4):356. doi: 10.1038/s41419-022-04834-5. Cell Death Dis. 2022. PMID: 35436993 Free PMC article.