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Liver-directed gene therapy for ornithine aminotransferase deficiency.
Boffa I, Polishchuk E, De Stefano L, Dell'Aquila F, Nusco E, Marrocco E, Audano M, Pedretti S, Caterino M, Bellezza I, Ruoppolo M, Mitro N, Cellini B, Auricchio A, Brunetti-Pierri N. Boffa I, et al. EMBO Mol Med. 2023 Apr 11;15(4):e17033. doi: 10.15252/emmm.202217033. Epub 2023 Jan 17. EMBO Mol Med. 2023. PMID: 36647689 Free PMC article.
Gene transfer of master autophagy regulator TFEB results in clearance of toxic protein and correction of hepatic disease in alpha-1-anti-trypsin deficiency.
Pastore N, Blomenkamp K, Annunziata F, Piccolo P, Mithbaokar P, Maria Sepe R, Vetrini F, Palmer D, Ng P, Polishchuk E, Iacobacci S, Polishchuk R, Teckman J, Ballabio A, Brunetti-Pierri N. Pastore N, et al. EMBO Mol Med. 2013 Mar;5(3):397-412. doi: 10.1002/emmm.201202046. Epub 2013 Feb 4. EMBO Mol Med. 2013. PMID: 23381957 Free PMC article.
Retinal transduction profiles by high-capacity viral vectors.
Puppo A, Cesi G, Marrocco E, Piccolo P, Jacca S, Shayakhmetov DM, Parks RJ, Davidson BL, Colloca S, Brunetti-Pierri N, Ng P, Donofrio G, Auricchio A. Puppo A, et al. Gene Ther. 2014 Oct;21(10):855-65. doi: 10.1038/gt.2014.57. Epub 2014 Jul 3. Gene Ther. 2014. PMID: 24989814 Free PMC article.
Prevalence of anti-adeno-associated virus serotype 8 neutralizing antibodies and arylsulfatase B cross-reactive immunologic material in mucopolysaccharidosis VI patient candidates for a gene therapy trial.
Ferla R, Claudiani P, Savarese M, Kozarsky K, Parini R, Scarpa M, Donati MA, Sorge G, Hopwood JJ, Parenti G, Fecarotta S, Nigro V, Sivri HS, Van Der Ploeg A, Andria G, Brunetti-Pierri N, Auricchio A. Ferla R, et al. Hum Gene Ther. 2015 Mar;26(3):145-52. doi: 10.1089/hum.2014.109. Hum Gene Ther. 2015. PMID: 25654180 Free PMC article.
Enhancement of hepatic autophagy increases ureagenesis and protects against hyperammonemia.
Soria LR, Allegri G, Melck D, Pastore N, Annunziata P, Paris D, Polishchuk E, Nusco E, Thöny B, Motta A, Häberle J, Ballabio A, Brunetti-Pierri N. Soria LR, et al. Proc Natl Acad Sci U S A. 2018 Jan 9;115(2):391-396. doi: 10.1073/pnas.1714670115. Epub 2017 Dec 26. Proc Natl Acad Sci U S A. 2018. PMID: 29279371 Free PMC article.
259 results