Alessandro Fraldi - Publications

Telethon Institute of Genetics and Medicine, Italy 
Lysosomal storage disease, autophagy, lysosome, gene therapy, molecular therapy

28 high-probability publications. We are testing a new system for linking publications to authors. You can help! If you notice any inaccuracies, please sign in and mark papers as correct or incorrect matches. If you identify any major omissions or other inaccuracies in the publication list, please let us know.

Year Citation  Score
2020 Intartaglia D, Giamundo G, Marrocco E, Maffia V, Salierno FG, Nusco E, Fraldi A, Conte I, Sorrentino NC. Retinal Degeneration in MPS-IIIA Mouse Model. Frontiers in Cell and Developmental Biology. 8: 132. PMID 32195255 DOI: 10.3389/fcell.2020.00132  0.8
2020 Monaco A, Maffia V, Sorrentino NC, Sambri I, Ezhova Y, Giuliano T, Cacace V, Nusco E, De Risi M, De Leonibus E, Schrader T, Klärner FG, Bitan G, Fraldi A. The Amyloid Inhibitor CLR01 Relieves Autophagy and Ameliorates Neuropathology in a Severe Lysosomal Storage Disease. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 32087148 DOI: 10.1016/j.ymthe.2020.02.005  0.8
2019 Sorrentino NC, Cacace V, De Risi M, Maffia V, Strollo S, Tedesco N, Nusco E, Romagnoli N, Ventrella D, Huang Y, Liu N, Kalled SL, Choi VW, De Leonibus E, Fraldi A. Enhancing the Therapeutic Potential of Sulfamidase for the Treatment of Mucopolysaccharidosis IIIA. Molecular Therapy. Methods & Clinical Development. 15: 333-342. PMID 31788497 DOI: 10.1016/j.omtm.2019.10.009  0.8
2018 Fraldi A, Serafini M, Sorrentino NC, Gentner B, Aiuti A, Bernardo ME. Gene therapy for mucopolysaccharidoses: in vivo and ex vivo approaches. Italian Journal of Pediatrics. 44: 130. PMID 30442177 DOI: 10.1186/s13052-018-0565-y  0.44
2016 Zito E, Fraldi A, Pepe S, Annunziata I, Kobinger G, Di Natale P, Ballabio A, Cosma MP. Sulfatase activities are regulated by the interaction of the sulfatase-modifying factor 1 with SUMF2. Embo Reports. 17: 1901. PMID 27909074 DOI: 10.15252/embr.201570010  0.8
2016 Sambri I, D'Alessio R, Ezhova Y, Giuliano T, Sorrentino NC, Cacace V, De Risi M, Cataldi M, Annunziato L, De Leonibus E, Fraldi A. Lysosomal dysfunction disrupts presynaptic maintenance and restoration of presynaptic function prevents neurodegeneration in lysosomal storage diseases. Embo Molecular Medicine. PMID 27881461 DOI: 10.15252/emmm.201606965  0.44
2016 Sorrentino NC, Fraldi A. Brain Targeting in MPS-IIIA. Pediatric Endocrinology Reviews : Per. 13: 630-8. PMID 27491210  0.44
2016 Fraldi A, Klein AD, Medina DL, Settembre C. Brain Disorders Due to Lysomal Dysfunction. Annual Review of Neuroscience. PMID 27090953 DOI: 10.1146/annurev-neuro-070815-014031  0.64
2015 Sorrentino NC, Maffia V, Strollo S, Cacace V, Romagnoli N, Manfredi A, Ventrella D, Dondi F, Barone F, Giunti M, Graham AR, Huang Y, Kalled SL, Auricchio A, Bacci ML, ... ... Fraldi A, et al. A comprehensive map of CNS transduction by eight recombinant adeno-associated virus serotypes upon cerebrospinal fluid administration in pigs. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 26639405 DOI: 10.1038/mt.2015.212  0.48
2015 Lambertini C, Ventrella D, Barone F, Sorrentino NC, Dondi F, Fraldi A, Giunti M, Surace EM, Bacci ML, Romagnoli N. Transdermal spinal catheter placement in piglets: Description and validation of the technique. Journal of Neuroscience Methods. 255: 17-21. PMID 26238729 DOI: 10.1016/j.jneumeth.2015.07.021  0.48
2014 Romagnoli N, Ventrella D, Giunti M, Dondi F, Sorrentino NC, Fraldi A, Surace EM, Bacci ML. Access to cerebrospinal fluid in piglets via the cisterna magna: optimization and description of the technique. Laboratory Animals. 48: 345-8. PMID 24968696 DOI: 10.1177/0023677214540881  0.48
2014 Tardieu M, Zérah M, Husson B, de Bournonville S, Deiva K, Adamsbaum C, Vincent F, Hocquemiller M, Broissand C, Furlan V, Ballabio A, Fraldi A, Crystal RG, Baugnon T, Roujeau T, et al. Intracerebral administration of adeno-associated viral vector serotype rh.10 carrying human SGSH and SUMF1 cDNAs in children with mucopolysaccharidosis type IIIA disease: results of a phase I/II trial. Human Gene Therapy. 25: 506-16. PMID 24524415 DOI: 10.1089/hum.2013.238  0.48
2014 Quintavalle C, Di Costanzo S, Zanca C, Tasset I, Fraldi A, Incoronato M, Mirabelli P, Monti M, Ballabio A, Pucci P, Cuervo AM, Condorelli G. Phosphorylation-regulated degradation of the tumor-suppressor form of PED by chaperone-mediated autophagy in lung cancer cells. Journal of Cellular Physiology. 229: 1359-68. PMID 24477641 DOI: 10.1002/jcp.24569  0.48
2013 Settembre C, Fraldi A, Medina DL, Ballabio A. Signals from the lysosome: a control centre for cellular clearance and energy metabolism. Nature Reviews. Molecular Cell Biology. 14: 283-96. PMID 23609508 DOI: 10.1038/nrm3565  0.8
2013 Sorrentino NC, D'Orsi L, Sambri I, Nusco E, Monaco C, Spampanato C, Polishchuk E, Saccone P, De Leonibus E, Ballabio A, Fraldi A. A highly secreted sulphamidase engineered to cross the blood-brain barrier corrects brain lesions of mice with mucopolysaccharidoses type IIIA. Embo Molecular Medicine. 5: 675-90. PMID 23568409 DOI: 10.1002/emmm.201202083  0.48
2013 Bachetti T, Chiesa S, Castagnola P, Bani D, Di Zanni E, Omenetti A, D'Osualdo A, Fraldi A, Ballabio A, Ravazzolo R, Martini A, Gattorno M, Ceccherini I. Autophagy contributes to inflammation in patients with TNFR-associated periodic syndrome (TRAPS). Annals of the Rheumatic Diseases. 72: 1044-52. PMID 23117241 DOI: 10.1136/annrheumdis-2012-201952  0.48
2012 de Pablo-Latorre R, Saide A, Polishhuck EV, Nusco E, Fraldi A, Ballabio A. Impaired parkin-mediated mitochondrial targeting to autophagosomes differentially contributes to tissue pathology in lysosomal storage diseases. Human Molecular Genetics. 21: 1770-81. PMID 22215441 DOI: 10.1093/hmg/ddr610  0.8
2011 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. Transcriptional activation of lysosomal exocytosis promotes cellular clearance. Developmental Cell. 21: 421-30. PMID 21889421 DOI: 10.1016/j.devcel.2011.07.016  0.48
2011 Spampanato C, De Leonibus E, Dama P, Gargiulo A, Fraldi A, Sorrentino NC, Russo F, Nusco E, Auricchio A, Surace EM, Ballabio A. Efficacy of a combined intracerebral and systemic gene delivery approach for the treatment of a severe lysosomal storage disorder. Molecular Therapy : the Journal of the American Society of Gene Therapy. 19: 860-9. PMID 21326216 DOI: 10.1038/mt.2010.299  0.48
2010 Fraldi A, Annunziata F, Lombardi A, Kaiser HJ, Medina DL, Spampanato C, Fedele AO, Polishchuk R, Sorrentino NC, Simons K, Ballabio A. Lysosomal fusion and SNARE function are impaired by cholesterol accumulation in lysosomal storage disorders. The Embo Journal. 29: 3607-20. PMID 20871593 DOI: 10.1038/emboj.2010.237  0.8
2008 Fraldi A, Zito E, Annunziata F, Lombardi A, Cozzolino M, Monti M, Spampanato C, Ballabio A, Pucci P, Sitia R, Cosma MP. Multistep, sequential control of the trafficking and function of the multiple sulfatase deficiency gene product, SUMF1 by PDI, ERGIC-53 and ERp44. Human Molecular Genetics. 17: 2610-21. PMID 18508857 DOI: 10.1093/hmg/ddn161  0.48
2008 Settembre C, Fraldi A, Rubinsztein DC, Ballabio A. Lysosomal storage diseases as disorders of autophagy. Autophagy. 4: 113-4. PMID 18000397  0.8
2008 Settembre C, Fraldi A, Jahreiss L, Spampanato C, Venturi C, Medina D, de Pablo R, Tacchetti C, Rubinsztein DC, Ballabio A. A block of autophagy in lysosomal storage disorders. Human Molecular Genetics. 17: 119-29. PMID 17913701 DOI: 10.1093/hmg/ddm289  0.8
2007 Fraldi A, Hemsley K, Crawley A, Lombardi A, Lau A, Sutherland L, Auricchio A, Ballabio A, Hopwood JJ. Functional correction of CNS lesions in an MPS-IIIA mouse model by intracerebral AAV-mediated delivery of sulfamidase and SUMF1 genes. Human Molecular Genetics. 16: 2693-702. PMID 17725987 DOI: 10.1093/hmg/ddm223  0.48
2007 Fraldi A, Biffi A, Lombardi A, Visigalli I, Pepe S, Settembre C, Nusco E, Auricchio A, Naldini L, Ballabio A, Cosma MP. SUMF1 enhances sulfatase activities in vivo in five sulfatase deficiencies. The Biochemical Journal. 403: 305-12. PMID 17206939 DOI: 10.1042/BJ20061783  0.48
2005 Dulac C, Michels AA, Fraldi A, Bonnet F, Nguyen VT, Napolitano G, Lania L, Bensaude O. Transcription-dependent association of multiple positive transcription elongation factor units to a HEXIM multimer. The Journal of Biological Chemistry. 280: 30619-29. PMID 15994294 DOI: 10.1074/jbc.M502471200  0.48
2005 Zito E, Fraldi A, Pepe S, Annunziata I, Kobinger G, Di Natale P, Ballabio A, Cosma MP. Sulphatase activities are regulated by the interaction of sulphatase-modifying factor 1 with SUMF2. Embo Reports. 6: 655-60. PMID 15962010 DOI: 10.1038/sj.embor.7400454  0.48
2004 Michels AA, Fraldi A, Li Q, Adamson TE, Bonnet F, Nguyen VT, Sedore SC, Price JP, Price DH, Lania L, Bensaude O. Binding of the 7SK snRNA turns the HEXIM1 protein into a P-TEFb (CDK9/cyclin T) inhibitor. The Embo Journal. 23: 2608-19. PMID 15201869 DOI: 10.1038/sj.emboj.7600275  0.48
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