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Transcription factor EB (TFEB) is a new therapeutic target for Pompe disease.
Spampanato C, Feeney E, Li L, Cardone M, Lim JA, Annunziata F, Zare H, Polishchuk R, Puertollano R, Parenti G, Ballabio A, Raben N. Spampanato C, et al. Among authors: polishchuk r. EMBO Mol Med. 2013 May;5(5):691-706. doi: 10.1002/emmm.201202176. Epub 2013 Apr 18. EMBO Mol Med. 2013. PMID: 23606558 Free PMC article.
Transcriptional activation of lysosomal exocytosis promotes cellular clearance.
Medina DL, Fraldi A, Bouche V, Annunziata F, Mansueto G, Spampanato C, Puri C, Pignata A, Martina JA, Sardiello M, Palmieri M, Polishchuk R, Puertollano R, Ballabio A. Medina DL, et al. Among authors: polishchuk r. Dev Cell. 2011 Sep 13;21(3):421-30. doi: 10.1016/j.devcel.2011.07.016. Epub 2011 Sep 1. Dev Cell. 2011. PMID: 21889421 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. Among authors: polishchuk r, polishchuk e. 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.
A gene network regulating lysosomal biogenesis and function.
Sardiello M, Palmieri M, di Ronza A, Medina DL, Valenza M, Gennarino VA, Di Malta C, Donaudy F, Embrione V, Polishchuk RS, Banfi S, Parenti G, Cattaneo E, Ballabio A. Sardiello M, et al. Science. 2009 Jul 24;325(5939):473-7. doi: 10.1126/science.1174447. Epub 2009 Jun 25. Science. 2009. PMID: 19556463
Wilson disease protein ATP7B utilizes lysosomal exocytosis to maintain copper homeostasis.
Polishchuk EV, Concilli M, Iacobacci S, Chesi G, Pastore N, Piccolo P, Paladino S, Baldantoni D, van IJzendoorn SC, Chan J, Chang CJ, Amoresano A, Pane F, Pucci P, Tarallo A, Parenti G, Brunetti-Pierri N, Settembre C, Ballabio A, Polishchuk RS. Polishchuk EV, et al. Dev Cell. 2014 Jun 23;29(6):686-700. doi: 10.1016/j.devcel.2014.04.033. Epub 2014 Jun 5. Dev Cell. 2014. PMID: 24909901 Free PMC article.
AAV-mediated transcription factor EB (TFEB) gene delivery ameliorates muscle pathology and function in the murine model of Pompe Disease.
Gatto F, Rossi B, Tarallo A, Polishchuk E, Polishchuk R, Carrella A, Nusco E, Alvino FG, Iacobellis F, De Leonibus E, Auricchio A, Diez-Roux G, Ballabio A, Parenti G. Gatto F, et al. Among authors: polishchuk r, polishchuk e. Sci Rep. 2017 Nov 8;7(1):15089. doi: 10.1038/s41598-017-15352-2. Sci Rep. 2017. PMID: 29118420 Free PMC article.
169 results