Alessandra Biffi

Summary

Affiliation: San Raffaele Scientific Institute
Country: Italy

Publications

  1. pmc Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells
    Alessandra Biffi
    San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scienctific Institute, Milan, Italy
    J Clin Invest 113:1118-29. 2004
  2. ncbi request reprint Gene therapy of storage disorders by retroviral and lentiviral vectors
    Alessandra Biffi
    San Raffaele Telethon Institute for Gene Therapy and Vita Salute University, H San Raffaele Scientific Institute, Milan 20132, Italy
    Hum Gene Ther 16:1133-42. 2005
  3. pmc Sulfatase modifying factor 1-mediated fibroblast growth factor signaling primes hematopoietic multilineage development
    Mario Buono
    Telethon Institute of Genetics and Medicine, 80134 Naples, Italy
    J Exp Med 207:1647-60. 2010
  4. ncbi request reprint Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy
    Alessandra Biffi
    San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute, 20132 Milan, Italy
    Science 341:1233158. 2013
  5. pmc Gene therapy of metachromatic leukodystrophy reverses neurological damage and deficits in mice
    Alessandra Biffi
    San Raffaele Telethon Institute for Gene Therapy, Vita Salute San Raffaele University, Milan, Italy
    J Clin Invest 116:3070-82. 2006
  6. ncbi request reprint Human hematopoietic stem cells in gene therapy: pre-clinical and clinical issues
    Alessandra Biffi
    San Raffaele Telethon Institute for Gene Therapy, H San Raffaele Scientific Institute, Milan, Italy
    Curr Gene Ther 8:135-46. 2008
  7. doi request reprint Development and maturation of invariant NKT cells in the presence of lysosomal engulfment
    Tiziana Plati
    San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute, 20132 Milan, Italy
    Eur J Immunol 39:2748-54. 2009
  8. pmc The galactocerebrosidase enzyme contributes to the maintenance of a functional hematopoietic stem cell niche
    Ilaria Visigalli
    San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute, Via Olgettina 58, Milan, Italy
    Blood 116:1857-66. 2010
  9. doi request reprint Absence of VOD in paediatric thalassaemic HSCT recipients using defibrotide prophylaxis and intravenous Busulphan
    Barbara Cappelli
    Pediatric Immunohematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Milan, Italy
    Br J Haematol 147:554-60. 2009
  10. doi request reprint Characterization of new arylsulfatase A gene mutations reinforces genotype-phenotype correlation in metachromatic leukodystrophy
    Martina Cesani
    San Raffaele Telethon Institute for Gene Therapy HSR TIGET, Milan, Italy
    Hum Mutat 30:E936-45. 2009

Collaborators

Detail Information

Publications29

  1. pmc Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells
    Alessandra Biffi
    San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scienctific Institute, Milan, Italy
    J Clin Invest 113:1118-29. 2004
    ..These results indicate that transplantation of LV-transduced autologous HSCs represents a potentially efficacious therapeutic strategy for MLD and possibly other neurodegenerative disorders...
  2. ncbi request reprint Gene therapy of storage disorders by retroviral and lentiviral vectors
    Alessandra Biffi
    San Raffaele Telethon Institute for Gene Therapy and Vita Salute University, H San Raffaele Scientific Institute, Milan 20132, Italy
    Hum Gene Ther 16:1133-42. 2005
  3. pmc Sulfatase modifying factor 1-mediated fibroblast growth factor signaling primes hematopoietic multilineage development
    Mario Buono
    Telethon Institute of Genetics and Medicine, 80134 Naples, Italy
    J Exp Med 207:1647-60. 2010
    ..These data indicate that Sumf1 controls HSPC differentiation and hematopoietic lineage development through FGF and Wnt signaling...
  4. ncbi request reprint Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy
    Alessandra Biffi
    San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute, 20132 Milan, Italy
    Science 341:1233158. 2013
    ..These findings indicate that extensive genetic engineering of human hematopoiesis can be achieved with lentiviral vectors and that this approach may offer therapeutic benefit for MLD patients. ..
  5. pmc Gene therapy of metachromatic leukodystrophy reverses neurological damage and deficits in mice
    Alessandra Biffi
    San Raffaele Telethon Institute for Gene Therapy, Vita Salute San Raffaele University, Milan, Italy
    J Clin Invest 116:3070-82. 2006
    ..Overall, our data provide a strong rationale for implementing HSPC gene therapy in MLD patients...
  6. ncbi request reprint Human hematopoietic stem cells in gene therapy: pre-clinical and clinical issues
    Alessandra Biffi
    San Raffaele Telethon Institute for Gene Therapy, H San Raffaele Scientific Institute, Milan, Italy
    Curr Gene Ther 8:135-46. 2008
    ....
  7. doi request reprint Development and maturation of invariant NKT cells in the presence of lysosomal engulfment
    Tiziana Plati
    San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute, 20132 Milan, Italy
    Eur J Immunol 39:2748-54. 2009
    ..Rather, lipid ligand(s) or storage compounds, which are affected in those LSD lacking mature iNKT cells, might indeed be relevant for iNKT cell selection...
  8. pmc The galactocerebrosidase enzyme contributes to the maintenance of a functional hematopoietic stem cell niche
    Ilaria Visigalli
    San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute, Via Olgettina 58, Milan, Italy
    Blood 116:1857-66. 2010
    ..Therefore, GALC and, possibly, other enzymes for the maintenance of niche functionality and health tightly control the concentration of these sphingolipids within HSPCs...
  9. doi request reprint Absence of VOD in paediatric thalassaemic HSCT recipients using defibrotide prophylaxis and intravenous Busulphan
    Barbara Cappelli
    Pediatric Immunohematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Milan, Italy
    Br J Haematol 147:554-60. 2009
    ..A galenic preparation of oral DF also permits this treatment in low-weight patients. Costs of DF prophylaxis are acceptable considering the reduced incidence of VOD...
  10. doi request reprint Characterization of new arylsulfatase A gene mutations reinforces genotype-phenotype correlation in metachromatic leukodystrophy
    Martina Cesani
    San Raffaele Telethon Institute for Gene Therapy HSR TIGET, Milan, Italy
    Hum Mutat 30:E936-45. 2009
    ....
  11. pmc Brain conditioning is instrumental for successful microglia reconstitution following hematopoietic stem cell transplantation
    Alessia Capotondo
    Division of Regenerative Medicine, Stem Cells and Gene Therapy, Vita Salute San Raffaele University, San Raffaele Scientific Institute, 20132 Milan, Italy
    Proc Natl Acad Sci U S A 109:15018-23. 2012
    ....
  12. pmc Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model
    Ilaria Visigalli
    San Raffaele Telethon Institute for Gene Therapy, Division of Regenerative Medicine, Stem Cells and Gene Therapy, Milan, Italy
    Blood 116:5130-9. 2010
    ..Overall, our data provide evidence of an efficacious treatment for MPS I Hurler patients, warranting future development toward clinical testing...
  13. ncbi request reprint Lentiviral vector common integration sites in preclinical models and a clinical trial reflect a benign integration bias and not oncogenic selection
    Alessandra Biffi
    San Raffaele Telethon Institute for Gene Therapy HSR TIGET, Via Olgettina 58, Milan, Italy
    Blood 117:5332-9. 2011
    ..These findings imply that LV CISs are produced by an integration bias toward specific genomic regions rather than by oncogenic selection...
  14. doi request reprint Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome
    Alessandro Aiuti
    San Raffaele Telethon Institute for Gene Therapy, Division of Regenerative Medicine, Stem Cells, and Gene Therapy, San Raffaele Scientific Institute, 20132 Milan, Italy
    Science 341:1233151. 2013
    ..Although extended clinical observation is required to establish long-term safety, lentiviral gene therapy represents a promising treatment for WAS. ..
  15. doi request reprint Identification of hematopoietic stem cell-specific miRNAs enables gene therapy of globoid cell leukodystrophy
    Bernhard Gentner
    San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute, Milan 20132, Italy
    Sci Transl Med 2:58ra84. 2010
    ..This approach protected HSCs from GALC toxicity and allowed successful treatment of a mouse GLD model, providing a rationale to explore HSC-based gene therapy for GLD...
  16. doi request reprint Unpredictability of intravenous busulfan pharmacokinetics in children undergoing hematopoietic stem cell transplantation for advanced beta thalassemia: limited toxicity with a dose-adjustment policy
    Robert Chiesa
    Pediatric Immunohematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Milan, Italy
    Biol Blood Marrow Transplant 16:622-8. 2010
    ..Conditioning with i.v. Bu and dose adjustment is feasible and well tolerated, although recurrence of thalassemia remains an unsolved problem in children with advanced disease...
  17. doi request reprint Fatal vancomycin- and linezolid-resistant Enterococcus faecium sepsis in a child undergoing allogeneic haematopoietic stem cell transplantation for beta-thalassaemia major
    Marco Fossati
    Pediatric Immunohematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Milan, Italy
    J Med Microbiol 59:839-42. 2010
    ....
  18. doi request reprint Gene therapy for leukodystrophies
    Alessandra Biffi
    San Raffaele Telethon Institute for Gene Therapy HSR TIGET, Division of Regenerative Medicine, Stem Cells and Gene Therapy, San Raffaele Scientific Institute, Milan, Italy
    Hum Mol Genet 20:R42-53. 2011
    ..The development of new gene therapy viral vectors allowing targeting of the disease gene into oligodendrocytes or astrocytes should soon benefit other forms of LDs...
  19. doi request reprint Metallothioneins as dynamic markers for brain disease in lysosomal disorders
    Martina Cesani
    San Raffaele Telethon Institute for Gene Therapy HSR TIGET, Division of Regenerative Medicine, Stem Cells and Gene Therapy, San Raffaele Scientific Institute, Milan, Italy Neurogenomics Laboratory, Harvard Medical School and Brigham and Women s Hospital, Cambridge, MA, USA
    Ann Neurol 75:127-37. 2014
    ..To this end, we sought to identify blood transcripts associated with the progression of MLD...
  20. doi request reprint Dlg1, Sec8, and Mtmr2 regulate membrane homeostasis in Schwann cell myelination
    Annalisa Bolis
    Dulbecco Telethon Institute, San Raffaele Scientific Institute, 20132 Milan, Italy
    J Neurosci 29:8858-70. 2009
    ..Myelin outfoldings thus arise as a consequence of the loss of negative control on the amount of membrane, which is produced during myelination...
  21. ncbi request reprint Monitoring disease evolution and treatment response in lysosomal disorders by the peripheral benzodiazepine receptor ligand PK11195
    Ilaria Visigalli
    San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute, Milan, Italy
    Neurobiol Dis 34:51-62. 2009
    ....
  22. ncbi request reprint Genetically-modified hematopoietic stem cells and their progeny for widespread and efficient protein delivery to diseased sites: the case of lysosomal storage disorders
    Alessandra Biffi
    San Raffaele Telethon Institute for Gene Therapy, Via Olgettina 58, 20132, Milano, Italy
    Curr Gene Ther 12:381-8. 2012
    ..Among these, we here review one of the most promising approaches based on hematopoietic stem cells, taking advantage of lysosomal storage disorders as representative disease setting...
  23. pmc Preclinical safety and efficacy of human CD34(+) cells transduced with lentiviral vector for the treatment of Wiskott-Aldrich syndrome
    Samantha Scaramuzza
    San Raffaele Telethon Institute for Gene Therapy, Milan, Italy
    Mol Ther 21:175-84. 2013
    ..In summary, this work establishes the preclinical safety and efficacy of human CD34(+) cells gene therapy for the treatment of WAS...
  24. doi request reprint Critical issues for the proper diagnosis of Metachromatic Leukodystrophy
    Laura Lorioli
    San Raffaele Telethon Institute for Gene Therapy HSR TIGET, Division of Regenerative Medicine, Stem Cells and Gene Therapy, San Raffaele Scientific Institute, Milan, Italy HSR TIGET Pediatric Clinical Research Unit, Division of Regenerative Medicine, Stem Cells and Gene Therapy, San Raffaele Scientific Institute, Milan, Italy Pediatric Immunohematology, San Raffaele Scientific Institute, Milan, Italy Vita Salute San Raffaele University, Milan, Italy
    Gene 537:348-51. 2014
    ..Only combination of gene sequencing with thorough biochemical analysis allowed the correct diagnosis of the sibling, who was promptly directed to treatment. ..
  25. ncbi request reprint Coordinate dual-gene transgenesis by lentiviral vectors carrying synthetic bidirectional promoters
    Mario Amendola
    San Raffaele Telethon Institute for Gene Therapy HSR TIGET, San Raffaele Scientific Institute, Via Olgettina 58, 20132 Milano, Italy
    Nat Biotechnol 23:108-16. 2005
    ..Vectors incorporating these promoters should increase the power of gene function studies and expand the reach and safety of gene therapy...
  26. ncbi request reprint Novel candidate disease for gene therapy: metachromatic leukodystrophy
    Alessandra Biffi
    San Raffaele Telethon Institute for Gene Therapy and Vita Salute University, H San Raffaele Scientific Institute, Milan, Italy a biffi hsr it
    Expert Opin Biol Ther 7:1193-205. 2007
    ..Some of the gene therapy approaches discussed here, such as hematopoietic stem cells gene therapy, are likely to enter clinical testing in the near future...
  27. doi request reprint Maintenance of a functional hematopoietic stem cell niche through galactocerebrosidase and other enzymes
    Ilaria Visigalli
    Division of Regenerative Medicine and Stem Cells, San Raffaele Telethon Institute for Gene Therapy HSR TIGET, San Raffaele Scientific Institute, Milan, Italy
    Curr Opin Hematol 18:214-9. 2011
    ..Several enzymes were described as essential for guaranteeing niche functionality. This review summarizes the recent findings about the role of galactocerebrosidase and other enzymes involved in the maintenance of a functional HSC niche...
  28. pmc 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
    Anna Noè
    Pediatric Immunohaematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Milan, Italy
    Ital J Pediatr 36:14. 2010
    ..Neurotoxicity is a recognized complication of cyclosporine A (CSA) treatment. The incidence of severe CSA-related neurological complications following hematopoietic stem cell transplantation (HSCT) is 4-11%...
  29. ncbi request reprint Somatic intragenic recombination of the arylsulfatase A gene in a metachromatic leukodystrophy patient
    Stefano Regis
    Diagnosi Pre Postnatale Malattie Metaboliche Laboratory IRCCS G Gaslini, Genova, Italy
    Mol Genet Metab 89:150-5. 2006
    ..Although the phenotype in this patient was not modified by the recombination, similar events could potentially yield significant clinical benefits...