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54 results

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Page 1
TFEB links autophagy to lysosomal biogenesis.
Settembre C, Di Malta C, Polito VA, Garcia Arencibia M, Vetrini F, Erdin S, Erdin SU, Huynh T, Medina D, Colella P, Sardiello M, Rubinsztein DC, Ballabio A. Settembre C, et al. Among authors: colella p. Science. 2011 Jun 17;332(6036):1429-33. doi: 10.1126/science.1204592. Epub 2011 May 26. Science. 2011. PMID: 21617040 Free PMC article.
Emerging Issues in AAV-Mediated In Vivo Gene Therapy.
Colella P, Ronzitti G, Mingozzi F. Colella P, et al. Mol Ther Methods Clin Dev. 2017 Dec 1;8:87-104. doi: 10.1016/j.omtm.2017.11.007. eCollection 2018 Mar 16. Mol Ther Methods Clin Dev. 2017. PMID: 29326962 Free PMC article. Review.
Successful treatment of severe MSUD in Bckdhb-/- mice with neonatal AAV gene therapy.
Pontoizeau C, Gaborit C, Tual N, Simon-Sola M, Rotaru I, Benoist M, Colella P, Lamazière A, Brassier A, Arnoux JB, Rötig A, Ottolenghi C, de Lonlay P, Mingozzi F, Cavazzana M, Schiff M. Pontoizeau C, et al. Among authors: colella p. J Inherit Metab Dis. 2024 Jan;47(1):41-49. doi: 10.1002/jimd.12604. Epub 2023 Mar 15. J Inherit Metab Dis. 2024. PMID: 36880392
Gene Therapy for Pompe Disease: The Time is now.
Colella P, Mingozzi F. Colella P, et al. Hum Gene Ther. 2019 Oct;30(10):1245-1262. doi: 10.1089/hum.2019.109. Epub 2019 Sep 9. Hum Gene Ther. 2019. PMID: 31298581 Review.
CNS Repopulation by Hematopoietic-Derived Microglia-Like Cells Corrects Progranulin deficiency.
Colella P, Sayana R, Suarez-Nieto MV, Sarno J, Nyame K, Xiong J, Vera LNP, Basurto JA, Corbo M, Limaye A, Davis KL, Abu-Remaileh M, Gomez-Ospina N. Colella P, et al. Res Sq [Preprint]. 2023 Sep 12:rs.3.rs-3263412. doi: 10.21203/rs.3.rs-3263412/v1. Res Sq. 2023. Update in: Nat Commun. 2024 Jul 5;15(1):5654. doi: 10.1038/s41467-024-49908-4 PMID: 37790525 Free PMC article. Updated. Preprint.
Neonatal gene therapy achieves sustained disease rescue of maple syrup urine disease in mice.
Pontoizeau C, Simon-Sola M, Gaborit C, Nguyen V, Rotaru I, Tual N, Colella P, Girard M, Biferi MG, Arnoux JB, Rötig A, Ottolenghi C, de Lonlay P, Mingozzi F, Cavazzana M, Schiff M. Pontoizeau C, et al. Among authors: colella p. Nat Commun. 2022 Jun 7;13(1):3278. doi: 10.1038/s41467-022-30880-w. Nat Commun. 2022. PMID: 35672312 Free PMC article.
54 results