V. Calbi

发表

The proteasome load versus capacity balance determines apoptotic sensitivity of multiple myeloma cells to proteasome inhibition.

K. Anderson, P. Cascio, R. Sitia, 2009, Blood.

Proteasome Stress Causes Apoptotic Sensitivity of Multiple Myeloma Cells to Proteasome Inhibition

K. Anderson, P. Cascio, D. Chauhan, 2008 .

Lentiviral haemopoietic stem/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1/2 clinical study

L. Biasco, S. Galimberti, M. Valsecchi, 2019, The Lancet. Haematology.

Toward Reference Intervals of ARSA Activity in the Cerebrospinal Fluid: Implication for the Clinical Practice of Metachromatic Leukodystrophy.

F. Morena, S. Martino, A. Biffi, 2020, The journal of applied laboratory medicine.

Gene therapy for lysosomal storage disorders: recent advances for metachromatic leukodystrophy and mucopolysaccaridosis I

A. Aiuti, M. Bernardo, V. Calbi, 2017, Journal of Inherited Metabolic Disease.

Unexpected Huge Prevalence of Intracardiac Extension of Wilms Tumor—A Single Center Experience from a Ugandan Hospital

M. Mapelli, P. Agostoni, M. Muratori, 2022, Children.

Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent ß-thalassemia

S. Perrotta, M. Lidonnici, S. Galimberti, 2019, Nature Medicine.

Distinct Viral and Mutational Spectrum of Endemic Burkitt Lymphoma

S. Pileri, R. Rabadán, P. Piccaluga, 2015, PLoS pathogens.

Use of Defibrotide to help prevent post-transplant endothelial injury in a genetically predisposed infant with metachromatic leukodystrophy undergoing hematopoietic stem cell gene therapy

G. Ardissino, L. Naldini, F. Morena, 2018, Bone Marrow Transplantation.

A Case of William’s Syndrome in a Ugandan Child: A Feasible Diagnosis Even in a Low-Resource Setting

M. Mapelli, P. Agostoni, V. Calbi, 2021, Children.

Peripheral blood stem and progenitor cell collection in pediatric candidates for ex vivo gene therapy: a 10-year series

V. Gallo, A. Aiuti, F. Ciceri, 2021, Molecular therapy. Methods & clinical development.

Case Report: Consistent disease manifestations with a staggered time course in two identical twins affected by adenosine deaminase 2 deficiency

A. Mortellaro, A. Aiuti, M. Ponzoni, 2022, Frontiers in Immunology.

Echocardiographic Screening for Rheumatic Heart Disease in a Ugandan Orphanage: Feasibility and Outcomes

L. Fusini, M. Mapelli, P. Agostoni, 2022, Children.

Bone marrow harvesting from paediatric patients undergoing haematopoietic stem cell gene therapy

A. Biffi, A. Aiuti, R. Miniero, 2019, Bone Marrow Transplantation.

Retrieval of vector integration sites from cell-free DNA

F. Bushman, A. Fischer, C. Klein, 2021, Nature Medicine.

Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome.

V. Gallo, S. Galimberti, M. Valsecchi, 2021, The New England journal of medicine.

Gene Therapy for Beta Thalassemia: Preliminary Results from the PHASE I/II Tiget-Bthal Trial of Autologous Hematopoietic Stem Cells Genetically Modified with GLOBE Lentiviral Vector

S. Perrotta, M. Lidonnici, L. Naldini, 2017 .

Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and expanded access

V. Gallo, P. Rancoita, C. Di Serio, 2022, The Lancet.

Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent ß-thalassemia

S. Perrotta, M. Lidonnici, S. Galimberti, 2019, Nature Medicine.

Clonality Analysis of Immunoglobulin Gene Rearrangement by Next-Generation Sequencing in Endemic Burkitt Lymphoma Suggests Antigen Drive Activation of BCR as Opposed to Sporadic Burkitt Lymphoma

S. Pileri, M. Iacono, P. Piccaluga, 2015, American journal of clinical pathology.

The Alteration of Lipid Metabolism in Burkitt Lymphoma Identifies a Novel Marker: Adipophilin

S. Pileri, K. Naresh, P. Piccaluga, 2012, PloS one.

Treatment outcome among children under-five years hospitalized with severe acute malnutrition in St. Mary’s hospital Lacor, Northern Uganda

V. Calbi, Richard Nyeko, Boniface Otto Ssegujja, 2016, BMC Nutrition.

Lymphomas in sub‐Saharan Africa – what can we learn and how can we help in improving diagnosis, managing patients and fostering translational research?

Ian Magrath, Lorenzo Leoncini, K. Naresh, 2011, British journal of haematology.

Gene expression analysis uncovers similarity and differences among Burkitt lymphoma subtypes.

S. Pileri, A. Califano, Manjunath Kustagi, 2010, Blood.

Bilateral proptosis as an early manifestation of juvenile myelomonocitic leukemia in an African child

V. Calbi, T. R. Okello, I. Pecorella, 2021 .

Leukocyte and Dried Blood Spot Arylsulfatase A Assay by Tandem Mass Spectrometry.

A. Vanderver, M. Gelb, J. Kurtzberg, 2020, Analytical chemistry.

Diagnosis of Burkitt lymphoma using an algorithmic approach – applicable in both resource‐poor and resource‐rich countries

K. Naresh, I. Magrath, L. Leoncini, 2011, British journal of haematology.

Emapalumab treatment in an ADA-SCID patient with refractory hemophagocytic lymphohistiocytosis- related graft failure and disseminated bacillus Calmette-Guérin infection

V. Gallo, C. Baldoli, A. Aiuti, 2020, Haematologica.

Wiskott–Aldrich syndrome: Oral findings and microbiota in children and review of the literature

V. Gallo, A. Aiuti, M. Bernardo, 2022, Clinical and experimental dental research.

Hemophagocytic inflammatory syndrome in ADA-SCID: report of two cases and literature review

V. Gallo, M. Duse, M. Bernardo, 2023, Frontiers in Immunology.

Metachromatic leukodystrophy: A single‐center longitudinal study of 45 patients

P. Rancoita, C. Di Serio, C. Baldoli, 2021, Journal of inherited metabolic disease.

Preserving Ambulation in a Gene Therapy-Treated Girl Affected by Metachromatic Leukodystrophy: A Case Report

C. Borghi, D. Pandarese, S. Faccioli, 2023, Journal of personalized medicine.

Mild SARS-CoV-2 Infection After Gene Therapy in a Child With Wiskott-Aldrich Syndrome: A Case Report

V. Gallo, L. Piemonti, A. Aiuti, 2020, Frontiers in Immunology.

Lentiviral hematopoietic stem and progenitor cell gene therapy provides durable clinical benefit in early-symptomatic early-juvenile metachromatic leukodystrophy

M. Filippi, V. Gallo, L. Naldini, 2021 .

Lentiviral hematopoietic stem and progenitor cell gene therapy (HSPC-GT) for metachromatic leukodystrophy (MLD): Clinical outcomes from 33 patients

M. Filippi, V. Gallo, L. Naldini, 2020 .

Long-term and real-world safety and efficacy of retroviral gene therapy for adenosine deaminase deficiency.

C. Cancrini, P. Rancoita, C. Di Serio, 2024, Nature medicine.

Newborn screening in metachromatic leukodystrophy - European consensus-based recommendations on clinical management.

V. Calbi, A. Zerem, Hanka Dekker, 2024, European journal of paediatric neurology : EJPN : official journal of the European Paediatric Neurology Society.

Inventory of current practices regarding hematopoietic stem cell transplantation in metachromatic leukodystrophy in Europe and neighboring countries

H. Graessner, V. Calbi, A. Zerem, 2024, Orphanet journal of rare diseases.

Early skeletal outcomes after hematopoietic stem and progenitor cell gene therapy for Hurler syndrome

M. Valsecchi, S. Pontesilli, C. Baldoli, 2024, Science Translational Medicine.