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Alessandra Biffi, M.D.


Publications listed below are automatically derived from MEDLINE/PubMed and other sources, which might result in incorrect or missing publications. Faculty can login to make corrections and additions.
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  1. Morena F, Argentati C, Acquati S, DeWall S, Kelly F, Calbi V, Fumagalli F, Zancan S, Biffi A, Aiuti A, Martino S. Toward Reference Intervals of ARSA Activity in the Cerebrospinal Fluid: Implication for the Clinical Practice of Metachromatic Leukodystrophy. J Appl Lab Med. 2021 Mar 01; 6(2):354-366. PMID: 32910180.
    Citations: 1     Fields:    
  2. Della Starza I, Nunes V, Lovisa F, Silvestri D, Cavalli M, Garofalo A, Campeggio M, De Novi LA, Soscia R, Oggioni C, Mussolin L, Biondi A, Guarini A, Valsecchi MG, Conter V, Biffi A, Basso G, Foà R, Cazzaniga G. Droplet Digital PCR Improves IG-/TR-based MRD Risk Definition in Childhood B-cell Precursor Acute Lymphoblastic Leukemia. Hemasphere. 2021 Mar; 5(3):e543. PMID: 33655199.
  3. d'Amore ESG, Mainardi C, Mussolin L, Carraro E, Alaggio R, Lazzari E, Fusetti S, Ghirotto C, Marzollo A, Biddeci G, Toffolutti T, Massano D, Scarzello G, Zuliani M, Putti MC, Biffi A, Pillon M. Histiocytic sarcoma arising in a child affected by Burkitt lymphoma, with t(8;14)(q24;q32) positivity in both tumors. Pediatr Hematol Oncol. 2021 Feb; 38(1):1-7. PMID: 33621154.
    Citations:    Fields:    
  4. Lovisa F, Garbin A, Crotti S, Di Battista P, Gallingani I, Damanti CC, Tosato A, Carraro E, Pillon M, Mafakheri E, Romanato F, Gaffo E, Biffi A, Bortoluzzi S, Agostini M, Mussolin L. Increased Tenascin C, Osteopontin and HSP90 Levels in Plasmatic Small Extracellular Vesicles of Pediatric ALK-Positive Anaplastic Large Cell Lymphoma: New Prognostic Biomarkers? Diagnostics (Basel). 2021 Feb 06; 11(2). PMID: 33562105.
  5. Garbin A, Lovisa F, Holmes AB, Damanti CC, Gallingani I, Carraro E, Accordi B, Veltri G, Pizzi M, d'Amore ESG, Pillon M, Biffi A, Basso K, Mussolin L. miR-939 acts as tumor suppressor by modulating JUNB transcriptional activity in pediatric anaplastic large cell lymphoma. Haematologica. 2021 Feb 01; 106(2):610-613. PMID: 32299901.
    Citations: 1     Fields:    
  6. Damanti CC, Gaffo E, Lovisa F, Garbin A, Di Battista P, Gallingani I, Tosato A, Pillon M, Carraro E, Mascarin M, Elia C, Biffi A, Bortoluzzi S, Mussolin L. MiR-26a-5p as a Reference to Normalize MicroRNA qRT-PCR Levels in Plasma Exosomes of Pediatric Hematological Malignancies. Cells. 2021 Jan 08; 10(1). PMID: 33429910.
    Citations:    Fields:    
  7. Styczynski J, Tridello G, Xhaard A, Medinger M, Mielke S, Taskinen M, Blijlevens N, Rodriguez MAB, Solano C, Nikolousis E, Biffi A, Groll AH, Junghanss C, Tsirigotis P, Lioure B, Šrámek J, Holler E, Galaverna F, Fagioli F, Knelange N, Wendel L, Gil L, de la Camara R, Mikulska M, Ljungman P. Use of letermovir in off-label indications: Infectious Diseases Working Party of European Society of Blood and Marrow Transplantation retrospective study. Bone Marrow Transplant. 2020 Dec 07. PMID: 33288863.
    Citations: 2     Fields:    
  8. Esrick EB, Lehmann LE, Biffi A, Achebe M, Brendel C, Ciuculescu MF, Daley H, MacKinnon B, Morris E, Federico A, Abriss D, Boardman K, Khelladi R, Shaw K, Negre H, Negre O, Nikiforow S, Ritz J, Pai SY, London WB, Dansereau C, Heeney MM, Armant M, Manis JP, Williams DA. Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease. N Engl J Med. 2021 01 21; 384(3):205-215. PMID: 33283990.
    Citations: 6     Fields:    Translation:HumansCellsCTClinical Trials
  9. Polo G, Gueraldi D, Giuliani A, Rubert L, Cazzorla C, Salviati L, Marzollo A, Biffi A, Burlina AP, Burlina AB. The combined use of enzyme activity and metabolite assays as a strategy for newborn screening of mucopolysaccharidosis type I. Clin Chem Lab Med. 2020 Nov 26; 58(12):2063-2072. PMID: 32432561.
    Citations: 5     Fields:    
  10. Peviani M, Spano G, Pagani A, Brugnara G, Covino C, Galli R, Biffi A, Politi LS. Lipophilic dye-compatible brain clearing technique allowing correlative magnetic resonance/high-resolution fluorescence imaging in rat models of glioblastoma. Sci Rep. 2020 10 21; 10(1):17974. PMID: 33087842.
    Citations:    Fields:    
  11. Biddeci G, Donà D, Geranio G, Spadini S, Petris MG, Pillon M, Biffi A, Putti MC. Systemic Fusariosis: A Rare Complication in Children with Acute Lymphoblastic Leukemia. J Fungi (Basel). 2020 Oct 09; 6(4). PMID: 33050258.
  12. Marzollo A, Maestrini G, La Starza R, Elia L, Malfona F, Pierini T, Tretti Parenzan C, Coppe A, Bortoluzzi S, Biffi A, Mecucci C, Bresolin S, Testi AM. A novel germline variant in PIK3R1 results in SHORT syndrome associated with TAL/LMO T-cell acute lymphoblastic leukemia. Am J Hematol. 2020 12; 95(12):E335-E338. PMID: 32936972.
    Citations:    Fields:    Translation:Humans
  13. Massano D, Cosma L, Garolla M, Sainati L, Biffi A. Hospital-based home care for children with cancer during the COVID-19 pandemic in northeastern Italy. Pediatr Blood Cancer. 2020 12; 67(12):e28501. PMID: 32881322.
    Citations:    Fields:    Translation:HumansCellsPHPublic Health
  14. Donà D, Giaquinto C, Baraldi E, Biffi A, Gamba P, Saieva AM, Antoniello L, Costenaro P, Masiero S, Sainati L, Da Dalt L, Perilongo G. COVID-19 Pandemic: Perspective of an Italian Tertiary Care Pediatric Center. Healthcare (Basel). 2020 Sep 01; 8(3). PMID: 32882820.
  15. Manara R, Dalla Torre A, Lucchetta M, Ermani M, Favaro A, Baracchini C, Favaretto S, Viaro F, Munaretto V, Sartori S, Ponticorvo S, Russo AG, Biffi A, Sainati L, Colombatti R. Visual cortex changes in children with sickle cell disease and normal visual acuity: a multimodal magnetic resonance imaging study. Br J Haematol. 2021 Jan; 192(1):151-157. PMID: 32789861.
    Citations:    Fields:    
  16. Zhu J, Eichler F, Biffi A, Duncan CN, Williams DA, Majzoub JA. The Changing Face of Adrenoleukodystrophy. Endocr Rev. 2020 08 01; 41(4). PMID: 32364223.
    Citations: 1     Fields:    
  17. Tremolada M, Taverna L, Bonichini S, Pillon M, Biffi A, Putti MC. Pediatric Patients Treated for Leukemia Back to School: A Mixed-Method Analysis of Narratives about Daily Life and Illness Experience. Behav Sci (Basel). 2020 Jul 01; 10(7). PMID: 32630265.
  18. Tremolada M, Taverna L, Bonichini S, Putti MC, Pillon M, Biffi A. Health Locus of Control in Parents of Children with Leukemia and Associations with Their Life Perceptions and Depression Symptomatology. Children (Basel). 2020 May 01; 7(5). PMID: 32369950.
    Citations: 1     
  19. Sainati L, Biffi A. How we deal with the COVID-19 epidemic in an Italian paediatric onco-haematology clinic located in a region with a high density of cases. Br J Haematol. 2020 05; 189(4):640-642. PMID: 32298461.
    Citations: 5     Fields:    Translation:HumansCellsPHPublic Health
  20. Pizzi M, Tazzoli S, Carraro E, Chaviano F, Massano D, Lovisa F, Mussolin L, Todesco A, Biffi A, d'Amore ES, Pillon M, Rugge M. Histology of pediatric classic Hodgkin lymphoma: From diagnosis to prognostic stratification. Pediatr Blood Cancer. 2020 05; 67(5):e28230. PMID: 32134194.
    Citations:    Fields:    Translation:HumansCellsCTClinical Trials
  21. Lovisa F, Di Battista P, Gaffo E, Damanti CC, Garbin A, Gallingani I, Carraro E, Pillon M, Biffi A, Bortoluzzi S, Mussolin L. RNY4 in Circulating Exosomes of Patients With Pediatric Anaplastic Large Cell Lymphoma: An Active Player? Front Oncol. 2020; 10:238. PMID: 32175280.
    Citations: 4     
  22. Mussolin L, Lovisa F, Gallingani I, Cavallaro E, Carraro E, Damanti CC, Vinti L, Sala A, Micalizzi C, Santoro N, Piglione M, Cellini M, Buffardi S, Buldini B, D'Amore ESG, Biffi A, Pillon M. Minimal residual disease analysis in childhood mature B-cell leukaemia/lymphoma treated with AIEOP LNH-97 protocol with/without anti-CD20 administration. Br J Haematol. 2020 05; 189(3):e108-e111. PMID: 32080837.
    Citations: 2     Fields:    Translation:Humans
  23. Tremolada M, Taverna L, Chiavetta IT, Bonichini S, Putti MC, Biffi A. Psychological Wellbeing in Adolescents with Leukaemia: A Comparative Study with Typical Development Peers. Int J Environ Res Public Health. 2020 01 16; 17(2). PMID: 31963108.
    Citations: 1     Fields:    Translation:Humans
  24. Biddeci G, Antonello M, Pizzi M, Petris MG, Pillon M, Donà D, Biffi A, Putti MC. Mucormycosis with peculiar aortic involvement in a child with acute lymphoblastic leukemia. Pediatr Hematol Oncol. 2020 Mar; 37(2):164-169. PMID: 31847684.
    Citations: 1     Fields:    Translation:Humans
  25. Mainardi C, Pizzi M, Marzollo A, Carraro E, Boaro MP, Mussolin L, Massano D, Tazzoli S, Onofrillo D, Cesaro S, Farruggia P, Putti MC, Alaggio R, Biffi A, d'Amore ESG, Pillon M. Pediatric IgG4-related lymphadenopathy: A rare condition associated with autoimmunity and lymphoproliferative disorders. Pediatr Allergy Immunol. 2020 04; 31(3):332-336. PMID: 31770465.
    Citations:    Fields:    Translation:Humans
  26. Molina Estevez FJ, Mathews TD, Biffi A, Peviani M. Simultaneous Flow Cytometric Characterization of Multiple Cell Types Retrieved from Mouse Brain/Spinal Cord Through Different Homogenization Methods. J Vis Exp. 2019 11 19; (153). PMID: 31814622.
    Citations:    Fields:    Translation:AnimalsCells
  27. Kim J, Hu C, Moufawad El Achkar C, Black LE, Douville J, Larson A, Pendergast MK, Goldkind SF, Lee EA, Kuniholm A, Soucy A, Vaze J, Belur NR, Fredriksen K, Stojkovska I, Tsytsykova A, Armant M, DiDonato RL, Choi J, Cornelissen L, Pereira LM, Augustine EF, Genetti CA, Dies K, Barton B, Williams L, Goodlett BD, Riley BL, Pasternak A, Berry ER, Pflock KA, Chu S, Reed C, Tyndall K, Agrawal PB, Beggs AH, Grant PE, Urion DK, Snyder RO, Waisbren SE, Poduri A, Park PJ, Patterson A, Biffi A, Mazzulli JR, Bodamer O, Berde CB, Yu TW. Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease. N Engl J Med. 2019 10 24; 381(17):1644-1652. PMID: 31597037.
    Citations: 48     Fields:    Translation:Humans
  28. Poletti V, Biffi A. Gene-Based Approaches to Inherited Neurometabolic Diseases. Hum Gene Ther. 2019 10; 30(10):1222-1235. PMID: 31397176.
    Citations: 4     Fields:    Translation:HumansAnimalsCells
  29. Page KM, Stenger EO, Connelly JA, Shyr D, West T, Wood S, Case L, Kester M, Shim S, Hammond L, Hammond M, Webb C, Biffi A, Bambach B, Fatemi A, Kurtzberg J. Hematopoietic Stem Cell Transplantation to Treat Leukodystrophies: Clinical Practice Guidelines from the Hunter's Hope Leukodystrophy Care Network. Biol Blood Marrow Transplant. 2019 12; 25(12):e363-e374. PMID: 31499213.
    Citations: 1     Fields:    Translation:Humans
  30. Tremolada M, Taverna L, Bonichini S, Pillon M, Biffi A. The Developmental Pathways of Preschool Children with Acute Lymphoblastic Leukemia: Communicative and Social Sequelae One Year after Treatment. Children (Basel). 2019 Aug 13; 6(8). PMID: 31412554.
  31. Pellin D, Loperfido M, Baricordi C, Wolock SL, Montepeloso A, Weinberg OK, Biffi A, Klein AM, Biasco L. A comprehensive single cell transcriptional landscape of human hematopoietic progenitors. Nat Commun. 2019 06 03; 10(1):2395. PMID: 31160568.
    Citations: 28     Fields:    Translation:HumansAnimalsCells
  32. Tucci F, Frittoli M, Barzaghi F, Calbi V, Migliavacca M, Ferrua F, Fumagalli F, Lorioli L, Castagnaro L, Facchini M, Fossati C, Zancan S, Massariello P, Manfredini M, Consiglieri G, Canarutto D, Recupero S, Calzatini F, Casiraghi M, Darin S, Antonioli G, Miniero R, Fiori R, Silvani P, Zambelli M, Marktel S, Gattillo S, Milani R, Santoleri L, Ciceri F, Biffi A, Cicalese MP, Bernardo ME, Aiuti A. Bone marrow harvesting from paediatric patients undergoing haematopoietic stem cell gene therapy. Bone Marrow Transplant. 2019 12; 54(12):1995-2003. PMID: 31150018.
    Citations: 1     Fields:    Translation:Humans
  33. Petrillo C, Calabria A, Piras F, Capotondo A, Spinozzi G, Cuccovillo I, Benedicenti F, Naldini L, Montini E, Biffi A, Gentner B, Kajaste-Rudnitski A. Assessing the Impact of Cyclosporin A on Lentiviral Transduction and Preservation of Human Hematopoietic Stem Cells in Clinically Relevant Ex Vivo Gene Therapy Settings. Hum Gene Ther. 2019 09; 30(9):1133-1146. PMID: 31037976.
    Citations: 4     Fields:    Translation:HumansAnimalsCells
  34. Squeri G, Passerini L, Ferro F, Laudisa C, Tomasoni D, Deodato F, Donati MA, Gasperini S, Aiuti A, Bernardo ME, Gentner B, Naldini L, Annoni A, Biffi A, Gregori S. Targeting a Pre-existing Anti-transgene T Cell Response for Effective Gene Therapy of MPS-I in the Mouse Model of the Disease. Mol Ther. 2019 07 03; 27(7):1215-1227. PMID: 31060789.
    Citations: 4     Fields:    Translation:HumansAnimalsCellsPHPublic Health
  35. Peviani M, Capasso Palmiero U, Cecere F, Milazzo R, Moscatelli D, Biffi A. Biodegradable polymeric nanoparticles administered in the cerebrospinal fluid: Brain biodistribution, preferential internalization in microglia and implications for cell-selective drug release. Biomaterials. 2019 07; 209:25-40. PMID: 31026609.
    Citations: 4     Fields:    Translation:AnimalsCells
  36. Ellison SM, Liao A, Wood S, Taylor J, Youshani AS, Rowlston S, Parker H, Armant M, Biffi A, Chan L, Farzaneh F, Wynn R, Jones SA, Heal P, Gaspar HB, Bigger BW. Pre-clinical Safety and Efficacy of Lentiviral Vector-Mediated Ex Vivo Stem Cell Gene Therapy for the Treatment of Mucopolysaccharidosis IIIA. Mol Ther Methods Clin Dev. 2019 Jun 14; 13:399-413. PMID: 31044143.
    Citations: 9     
  37. Wu Y, Zeng J, Roscoe BP, Liu P, Yao Q, Lazzarotto CR, Clement K, Cole MA, Luk K, Baricordi C, Shen AH, Ren C, Esrick EB, Manis JP, Dorfman DM, Williams DA, Biffi A, Brugnara C, Biasco L, Brendel C, Pinello L, Tsai SQ, Wolfe SA, Bauer DE. Highly efficient therapeutic gene editing of human hematopoietic stem cells. Nat Med. 2019 05; 25(5):776-783. PMID: 30911135.
    Citations: 65     Fields:    Translation:HumansCells
  38. Esrick EB, Manis JP, Daley H, Baricordi C, Trébéden-Negre H, Pierciey FJ, Armant M, Nikiforow S, Heeney MM, London WB, Biasco L, Asmal M, Williams DA, Biffi A. Successful hematopoietic stem cell mobilization and apheresis collection using plerixafor alone in sickle cell patients. Blood Adv. 2018 10 09; 2(19):2505-2512. PMID: 30282642.
    Citations: 7     Fields:    Translation:HumansCells
  39. Politi LS, Salsano E, Biffi A. Metachromatic Leukodystrophy: Too Frequent (Mis)Diagnosis? JAMA Neurol. 2018 08 01; 75(8):1027. PMID: 29913002.
    Citations:    Fields:    Translation:Humans
  40. Ben Nasr M, D'Addio F, Malvandi AM, Faravelli S, Castillo-Leon E, Usuelli V, Rocchio F, Letizia T, El Essawy AB, Assi E, Mameli C, Giani E, Macedoni M, Maestroni A, Dassano A, Loretelli C, Paroni M, Cannalire G, Biasucci G, Sala M, Biffi A, Zuccotti GV, Fiorina P. Prostaglandin E2 Stimulates the Expansion of Regulatory Hematopoietic Stem and Progenitor Cells in Type 1 Diabetes. Front Immunol. 2018; 9:1387. PMID: 29971065.
    Citations: 3     Fields:    
  41. Gatti S, Agostini A, Palmiero UC, Colombo C, Peviani M, Biffi A, Moscatelli D. Hydrazone linked doxorubicin-PLA prodrug nanoparticles with high drug loading. Nanotechnology. 2018 Jul 27; 29(30):305602. PMID: 29701613.
    Citations: 1     Fields:    Translation:AnimalsCells
  42. Biffi A. Gene Therapy as a Curative Option for ß-Thalassemia. N Engl J Med. 2018 04 19; 378(16):1551-1552. PMID: 29669229.
    Citations: 4     Fields:    Translation:HumansCells
  43. Cavalca E, Cesani M, Gifford JC, Sena-Esteves M, Terreni MR, Leoncini G, Peviani M, Biffi A. Metallothioneins are neuroprotective agents in lysosomal storage disorders. Ann Neurol. 2018 02; 83(2):418-432. PMID: 29369397.
    Citations: 3     Fields:    Translation:HumansAnimals
  44. Capotondo A, Milazzo R, Garcia-Manteiga JM, Cavalca E, Montepeloso A, Garrison BS, Peviani M, Rossi DJ, Biffi A. Intracerebroventricular delivery of hematopoietic progenitors results in rapid and robust engraftment of microglia-like cells. Sci Adv. 2017 12; 3(12):e1701211. PMID: 29226242.
    Citations: 5     Fields:    Translation:HumansAnimalsCells
  45. Gong Y, Sasidharan N, Laheji F, Frosch M, Musolino P, Tanzi R, Kim DY, Biffi A, El Khoury J, Eichler F. Microglial dysfunction as a key pathological change in adrenomyeloneuropathy. Ann Neurol. 2017 Nov; 82(5):813-827. PMID: 29059709.
    Citations: 7     Fields:    Translation:HumansAnimalsCells
  46. Biffi A. Hematopoietic Gene Therapies for Metabolic and Neurologic Diseases. Hematol Oncol Clin North Am. 2017 10; 31(5):869-881. PMID: 28895853.
    Citations: 3     Fields:    Translation:HumansAnimalsCells
  47. Biffi A. Hematopoietic Stem Cell Gene Therapy for Storage Disease: Current and New Indications. Mol Ther. 2017 05 03; 25(5):1155-1162. PMID: 28389320.
    Citations: 18     Fields:    Translation:HumansAnimalsCells
  48. Scaramuzza S, Biasco L, Ripamonti A, Castiello MC, Loperfido M, Draghici E, Hernandez RJ, Benedicenti F, Radrizzani M, Salomoni M, Ranzani M, Bartholomae CC, Vicenzi E, Finocchi A, Bredius R, Bosticardo M, Schmidt M, von Kalle C, Montini E, Biffi A, Roncarolo MG, Naldini L, Villa A, Aiuti A. Preclinical Safety and Efficacy of Human CD34+ Cells Transduced With Lentiviral Vector for the Treatment of Wiskott-Aldrich Syndrome. Mol Ther. 2013 Jan; 21(1):175-184. PMID: 28178602.
  49. Visigalli I, Delai S, Ferro F, Cecere F, Vezzoli M, Sanvito F, Chanut F, Benedicenti F, Spinozzi G, Wynn R, Calabria A, Naldini L, Montini E, Cristofori P, Biffi A. Preclinical Testing of the Safety and Tolerability of Lentiviral Vector-Mediated Above-Normal Alpha-L-Iduronidase Expression in Murine and Human Hematopoietic Cells Using Toxicology and Biodistribution Good Laboratory Practice Studies. Hum Gene Ther. 2016 10; 27(10):813-829. PMID: 27431943.
    Citations: 17     Fields:    Translation:HumansAnimalsCells
  50. Sessa M, Lorioli L, Fumagalli F, Acquati S, Redaelli D, Baldoli C, Canale S, Lopez ID, Morena F, Calabria A, Fiori R, Silvani P, Rancoita PM, Gabaldo M, Benedicenti F, Antonioli G, Assanelli A, Cicalese MP, Del Carro U, Sora MG, Martino S, Quattrini A, Montini E, Di Serio C, Ciceri F, Roncarolo MG, Aiuti A, Naldini L, Biffi A. Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial. Lancet. 2016 Jul 30; 388(10043):476-87. PMID: 27289174.
    Citations: 98     Fields:    Translation:HumansCellsCTClinical Trials
  51. Migliavacca M, Assanelli A, Ferrua F, Cicalese MP, Biffi A, Frittoli M, Silvani P, Chidini G, Calderini E, Mandelli A, Camporesi A, Milani R, Farinelli G, Nicoletti R, Ciceri F, Aiuti A, Bernardo ME. Pioglitazone as a novel therapeutic approach in chronic granulomatous disease. J Allergy Clin Immunol. 2016 06; 137(6):1913-1915.e2. PMID: 27056268.
    Citations: 8     Fields:    Translation:HumansCTClinical Trials
  52. Amit I, Baker D, Barker R, Berger B, Bertozzi C, Bhatia S, Biffi A, Demichelis F, Doudna J, Dowdy SF, Endy D, Helmstaedter M, Junca H, June C, Kamb S, Khvorova A, Kim DH, Kim JS, Krishnan Y, Lakadamyali M, Lappalainen T, Lewin S, Liao J, Loman N, Lundberg E, Lynd L, Martin C, Mellman I, Miyawaki A, Mummery C, Nelson K, Paz J, Peralta-Yahya P, Picotti P, Polyak K, Prather K, Qin J, Quake S, Regev A, Rogers JA, Shetty R, Sommer M, Stevens M, Stolovitzky G, Takahashi M, Tang F, Teichmann S, Torres-Padilla ME, Tripathi L, Vemula P, Verdine G, Vollmer F, Wang J, Ying JY, Zhang F, Zhang T. Voices of biotech. Nat Biotechnol. 2016 Mar; 34(3):270-5. PMID: 26963549.
    Citations: 2     Fields:    Translation:Humans
  53. Biffi A. Gene therapy for lysosomal storage disorders: a good start. Hum Mol Genet. 2016 Apr 15; 25(R1):R65-75. PMID: 26604151.
    Citations: 16     Fields:    Translation:Humans
  54. Cesani M, Lorioli L, Grossi S, Amico G, Fumagalli F, Spiga I, Filocamo M, Biffi A. Mutation Update of ARSA and PSAP Genes Causing Metachromatic Leukodystrophy. Hum Mutat. 2016 Jan; 37(1):16-27. PMID: 26462614.
    Citations: 21     Fields:    Translation:Humans
  55. Ungari S, Montepeloso A, Morena F, Cocchiarella F, Recchia A, Martino S, Gentner B, Naldini L, Biffi A. Design of a regulated lentiviral vector for hematopoietic stem cell gene therapy of globoid cell leukodystrophy. Mol Ther Methods Clin Dev. 2015; 2:15038. PMID: 26509184.
    Citations: 10     
  56. Domi T, Porrello E, Velardo D, Capotondo A, Biffi A, Tonlorenzi R, Amadio S, Ambrosi A, Miyagoe-Suzuki Y, Takeda S, Ruegg MA, Previtali SC. Mesoangioblast delivery of miniagrin ameliorates murine model of merosin-deficient congenital muscular dystrophy type 1A. Skelet Muscle. 2015; 5:30. PMID: 26347253.
    Citations: 6     Fields:    
  57. Biffi A. Clinical translation of TALENS: Treating SCID-X1 by gene editing in iPSCs. Cell Stem Cell. 2015 Apr 02; 16(4):348-9. PMID: 25842973.
    Citations: 4     Fields:    Translation:HumansCells
  58. Ricca A, Rufo N, Ungari S, Morena F, Martino S, Kulik W, Alberizzi V, Bolino A, Bianchi F, Del Carro U, Biffi A, Gritti A. Combined gene/cell therapies provide long-term and pervasive rescue of multiple pathological symptoms in a murine model of globoid cell leukodystrophy. Hum Mol Genet. 2015 Jun 15; 24(12):3372-89. PMID: 25749991.
    Citations: 26     Fields:    Translation:HumansAnimalsCells
  59. Cianciaruso C, Pagani A, Martelli C, Bacigaluppi M, Squadrito ML, Lo Dico A, De Palma M, Furlan R, Lucignani G, Falini A, Biffi A, Ottobrini L, Politi LS. Cellular magnetic resonance with iron oxide nanoparticles: long-term persistence of SPIO signal in the CNS after transplanted cell death. Nanomedicine (Lond). 2014 Jul; 9(10):1457-74. PMID: 24823433.
    Citations: 12     Fields:    Translation:AnimalsCells
  60. Cesani M, Cavalca E, Macco R, Leoncini G, Terreni MR, Lorioli L, Furlan R, Comi G, Doglioni C, Zacchetti D, Sessa M, Scherzer CR, Biffi A. Metallothioneins as dynamic markers for brain disease in lysosomal disorders. Ann Neurol. 2014 Jan; 75(1):127-37. PMID: 24242821.
    Citations: 7     Fields:    Translation:HumansAnimalsCells
  61. Lorioli L, Cesani M, Regis S, Morena F, Grossi S, Fumagalli F, Acquati S, Redaelli D, Pini A, Sessa M, Martino S, Filocamo M, Biffi A. Critical issues for the proper diagnosis of Metachromatic Leukodystrophy. Gene. 2014 Mar 10; 537(2):348-51. PMID: 24334127.
    Citations: 7     Fields:    Translation:Humans
  62. Morena F, di Girolamo I, Emiliani C, Gritti A, Biffi A, Martino S. A new analytical bench assay for the determination of arylsulfatase a activity toward galactosyl-3-sulfate ceramide: implication for metachromatic leukodystrophy diagnosis. Anal Chem. 2014 Jan 07; 86(1):473-81. PMID: 24294900.
    Citations: 7     Fields:    Translation:HumansAnimalsCells
  63. Aiuti A, Biasco L, Scaramuzza S, Ferrua F, Cicalese MP, Baricordi C, Dionisio F, Calabria A, Giannelli S, Castiello MC, Bosticardo M, Evangelio C, Assanelli A, Casiraghi M, Di Nunzio S, Callegaro L, Benati C, Rizzardi P, Pellin D, Di Serio C, Schmidt M, Von Kalle C, Gardner J, Mehta N, Neduva V, Dow DJ, Galy A, Miniero R, Finocchi A, Metin A, Banerjee PP, Orange JS, Galimberti S, Valsecchi MG, Biffi A, Montini E, Villa A, Ciceri F, Roncarolo MG, Naldini L. Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome. Science. 2013 Aug 23; 341(6148):1233151. PMID: 23845947.
    Citations: 386     Fields:    Translation:HumansCellsCTClinical Trials
  64. Biffi A, Montini E, Lorioli L, Cesani M, Fumagalli F, Plati T, Baldoli C, Martino S, Calabria A, Canale S, Benedicenti F, Vallanti G, Biasco L, Leo S, Kabbara N, Zanetti G, Rizzo WB, Mehta NA, Cicalese MP, Casiraghi M, Boelens JJ, Del Carro U, Dow DJ, Schmidt M, Assanelli A, Neduva V, Di Serio C, Stupka E, Gardner J, von Kalle C, Bordignon C, Ciceri F, Rovelli A, Roncarolo MG, Aiuti A, Sessa M, Naldini L. Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy. Science. 2013 Aug 23; 341(6148):1233158. PMID: 23845948.
    Citations: 409     Fields:    Translation:HumansCellsCTClinical Trials
  65. Biffi A. Genetically-modified hematopoietic stem cells and their progeny for widespread and efficient protein delivery to diseased sites: the case of lysosomal storage disorders. Curr Gene Ther. 2012 Oct; 12(5):381-8. PMID: 22934618.
    Citations: 4     Fields:    Translation:HumansAnimalsCells
  66. Capotondo A, Milazzo R, Politi LS, Quattrini A, Palini A, Plati T, Merella S, Nonis A, di Serio C, Montini E, Naldini L, Biffi A. Brain conditioning is instrumental for successful microglia reconstitution following hematopoietic stem cell transplantation. Proc Natl Acad Sci U S A. 2012 Sep 11; 109(37):15018-23. PMID: 22923692.
    Citations: 54     Fields:    Translation:AnimalsCells
  67. Scaramuzza S, Biasco L, Ripamonti A, Castiello MC, Loperfido M, Draghici E, Hernandez RJ, Benedicenti F, Radrizzani M, Salomoni M, Ranzani M, Bartholomae CC, Vicenzi E, Finocchi A, Bredius R, Bosticardo M, Schmidt M, von Kalle C, Montini E, Biffi A, Roncarolo MG, Naldini L, Villa A, Aiuti A. Preclinical safety and efficacy of human CD34(+) cells transduced with lentiviral vector for the treatment of Wiskott-Aldrich syndrome. Mol Ther. 2013 Jan; 21(1):175-84. PMID: 22371846.
    Citations: 38     Fields:    Translation:AnimalsCells
  68. Visigalli I, Biffi A. Maintenance of a functional hematopoietic stem cell niche through galactocerebrosidase and other enzymes. Curr Opin Hematol. 2011 Jul; 18(4):214-9. PMID: 21537167.
    Citations: 1     Fields:    Translation:HumansAnimalsCells
  69. Biffi A, Aubourg P, Cartier N. Gene therapy for leukodystrophies. Hum Mol Genet. 2011 Apr 15; 20(R1):R42-53. PMID: 21459776.
    Citations: 25     Fields:    Translation:HumansCells
  70. Biffi A, Bartolomae CC, Cesana D, Cartier N, Aubourg P, Ranzani M, Cesani M, Benedicenti F, Plati T, Rubagotti E, Merella S, Capotondo A, Sgualdino J, Zanetti G, von Kalle C, Schmidt M, Naldini L, Montini E. Lentiviral vector common integration sites in preclinical models and a clinical trial reflect a benign integration bias and not oncogenic selection. Blood. 2011 May 19; 117(20):5332-9. PMID: 21403130.
    Citations: 94     Fields:    Translation:HumansAnimalsCells
  71. Gentner B, Visigalli I, Hiramatsu H, Lechman E, Ungari S, Giustacchini A, Schira G, Amendola M, Quattrini A, Martino S, Orlacchio A, Dick JE, Biffi A, Naldini L. Identification of hematopoietic stem cell-specific miRNAs enables gene therapy of globoid cell leukodystrophy. Sci Transl Med. 2010 Nov 17; 2(58):58ra84. PMID: 21084719.
    Citations: 81     Fields:    Translation:HumansAnimalsCells
  72. Visigalli I, Delai S, Politi LS, Di Domenico C, Cerri F, Mrak E, D'Isa R, Ungaro D, Stok M, Sanvito F, Mariani E, Staszewsky L, Godi C, Russo I, Cecere F, Del Carro U, Rubinacci A, Brambilla R, Quattrini A, Di Natale P, Ponder K, Naldini L, Biffi A. Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model. Blood. 2010 Dec 09; 116(24):5130-9. PMID: 20847202.
    Citations: 67     Fields:    Translation:AnimalsCells
  73. Buono M, Visigalli I, Bergamasco R, Biffi A, Cosma MP. Sulfatase modifying factor 1-mediated fibroblast growth factor signaling primes hematopoietic multilineage development. J Exp Med. 2010 Aug 02; 207(8):1647-60. PMID: 20643830.
    Citations: 10     Fields:    Translation:AnimalsCells
  74. Visigalli I, Ungari S, Martino S, Park H, Cesani M, Gentner B, Sergi Sergi L, Orlacchio A, Naldini L, Biffi A. The galactocerebrosidase enzyme contributes to the maintenance of a functional hematopoietic stem cell niche. Blood. 2010 Sep 16; 116(11):1857-66. PMID: 20511539.
    Citations: 15     Fields:    Translation:HumansAnimalsCells
  75. Fossati M, Cappelli B, Biral E, Chiesa R, Biffi A, Ossi C, Moro M, Cirillo DM, Clementi M, Soliman C, Ciceri F, Roncarolo MG, Fumagalli L, Marktel S. Fatal vancomycin- and linezolid-resistant Enterococcus faecium sepsis in a child undergoing allogeneic haematopoietic stem cell transplantation for beta-thalassaemia major. J Med Microbiol. 2010 Jul; 59(Pt 7):839-842. PMID: 20299507.
    Citations: 1     Fields:    Translation:HumansCells
  76. Noè A, Cappelli B, Biffi A, Chiesa R, Frugnoli I, Biral E, Finizio V, Baldoli C, Vezzulli P, Minicucci F, Fanelli G, Fiori R, Ciceri F, Roncarolo MG, Marktel S. High incidence of severe cyclosporine neurotoxicity in children affected by haemoglobinopaties undergoing myeloablative haematopoietic stem cell transplantation: early diagnosis and prompt intervention ameliorates neurological outcome. Ital J Pediatr. 2010 Feb 06; 36:14. PMID: 20181110.
    Citations: 15     Fields:    Translation:Humans
  77. Chiesa R, Cappelli B, Crocchiolo R, Frugnoli I, Biral E, Noè A, Evangelio C, Fossati M, Roccia T, Biffi A, Finizio V, Aiuti A, Broglia M, Bartoli A, Ciceri F, Roncarolo MG, Marktel S. Unpredictability of intravenous busulfan pharmacokinetics in children undergoing hematopoietic stem cell transplantation for advanced beta thalassemia: limited toxicity with a dose-adjustment policy. Biol Blood Marrow Transplant. 2010 May; 16(5):622-8. PMID: 19963071.
    Citations: 9     Fields:    Translation:HumansCTClinical Trials
  78. Cesani M, Capotondo A, Plati T, Sergi LS, Fumagalli F, Roncarolo MG, Naldini L, Comi G, Sessa M, Biffi A. Characterization of new arylsulfatase A gene mutations reinforces genotype-phenotype correlation in metachromatic leukodystrophy. Hum Mutat. 2009 Oct; 30(10):E936-45. PMID: 19606494.
    Citations: 7     Fields:    Translation:HumansAnimalsCells
  79. Plati T, Visigalli I, Capotondo A, Buono M, Naldini L, Cosma MP, Biffi A. Development and maturation of invariant NKT cells in the presence of lysosomal engulfment. Eur J Immunol. 2009 Oct; 39(10):2748-54. PMID: 19637231.
    Citations: 6     Fields:    Translation:AnimalsCells
  80. Cappelli B, Chiesa R, Evangelio C, Biffi A, Roccia T, Frugnoli I, Biral E, Noè A, Fossati M, Finizio V, Miniero R, Napolitano S, Ferrua F, Soliman C, Ciceri F, Roncarolo MG, Marktel S. Absence of VOD in paediatric thalassaemic HSCT recipients using defibrotide prophylaxis and intravenous Busulphan. Br J Haematol. 2009 Nov; 147(4):554-60. PMID: 19747363.
    Citations: 6     Fields:    Translation:Humans
  81. Bolis A, Coviello S, Visigalli I, Taveggia C, Bachi A, Chishti AH, Hanada T, Quattrini A, Previtali SC, Biffi A, Bolino A. Dlg1, Sec8, and Mtmr2 regulate membrane homeostasis in Schwann cell myelination. J Neurosci. 2009 Jul 08; 29(27):8858-70. PMID: 19587293.
    Citations: 40     Fields:    Translation:HumansAnimalsCells
  82. Visigalli I, Moresco RM, Belloli S, Politi LS, Gritti A, Ungaro D, Matarrese M, Turolla E, Falini A, Scotti G, Naldini L, Fazio F, Biffi A. Monitoring disease evolution and treatment response in lysosomal disorders by the peripheral benzodiazepine receptor ligand PK11195. Neurobiol Dis. 2009 Apr; 34(1):51-62. PMID: 19320046.
    Citations: 6     Fields:    Translation:AnimalsCells
  83. Martino S, Tiribuzi R, Tortori A, Conti D, Visigalli I, Lattanzi A, Biffi A, Gritti A, Orlacchio A. Specific determination of beta-galactocerebrosidase activity via competitive inhibition of beta-galactosidase. Clin Chem. 2009 Mar; 55(3):541-8. PMID: 19147730.
    Citations: 19     Fields:    Translation:HumansAnimalsCells
  84. Biffi A, Cesani M. Human hematopoietic stem cells in gene therapy: pre-clinical and clinical issues. Curr Gene Ther. 2008 Apr; 8(2):135-46. PMID: 18393833.
    Citations: 8     Fields:    Translation:HumansCells
  85. Biffi A, Naldini L. Novel candidate disease for gene therapy: metachromatic leukodystrophy. Expert Opin Biol Ther. 2007 Aug; 7(8):1193-205. PMID: 17696818.
    Citations: 4     Fields:    Translation:HumansAnimals
  86. 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. Biochem J. 2007 Apr 15; 403(2):305-12. PMID: 17206939.
    Citations: 25     Fields:    Translation:HumansAnimalsCells
  87. Biffi A, Capotondo A, Fasano S, del Carro U, Marchesini S, Azuma H, Malaguti MC, Amadio S, Brambilla R, Grompe M, Bordignon C, Quattrini A, Naldini L. Gene therapy of metachromatic leukodystrophy reverses neurological damage and deficits in mice. J Clin Invest. 2006 Nov; 116(11):3070-82. PMID: 17080200.
    Citations: 67     Fields:    Translation:AnimalsCells
  88. Regis S, Lualdi S, Biffi A, Sessa M, Corsolini F, Parenti G, Filocamo M. Somatic intragenic recombination of the arylsulfatase A gene in a metachromatic leukodystrophy patient. Mol Genet Metab. 2006 Sep-Oct; 89(1-2):150-5. PMID: 16782379.
    Citations: 1     Fields:    Translation:HumansCells
  89. Biffi A, Naldini L. Gene therapy of storage disorders by retroviral and lentiviral vectors. Hum Gene Ther. 2005 Oct; 16(10):1133-42. PMID: 16218774.
    Citations: 8     Fields:    Translation:HumansAnimalsCells
  90. Amendola M, Venneri MA, Biffi A, Vigna E, Naldini L. Coordinate dual-gene transgenesis by lentiviral vectors carrying synthetic bidirectional promoters. Nat Biotechnol. 2005 Jan; 23(1):108-16. PMID: 15619618.
    Citations: 118     Fields:    Translation:HumansCells
  91. Biffi A, De Palma M, Quattrini A, Del Carro U, Amadio S, Visigalli I, Sessa M, Fasano S, Brambilla R, Marchesini S, Bordignon C, Naldini L. Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells. J Clin Invest. 2004 Apr; 113(8):1118-29. PMID: 15085191.
    Citations: 82     Fields:    Translation:AnimalsCells
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Funded by the NIH National Center for Advancing Translational Sciences through its Clinical and Translational Science Awards Program, grant number UL1TR002541.