A5082368982- CAR-T cell therapy research
- Hematopoietic Stem Cell Transplantation
- Immune Cell Function and Interaction
- Acute Lymphoblastic Leukemia research
- Immunodeficiency and Autoimmune Disorders
- Virus-based gene therapy research
- T-cell and B-cell Immunology
- Acute Myeloid Leukemia Research
- Cytomegalovirus and herpesvirus research
- CRISPR and Genetic Engineering
- Chronic Myeloid Leukemia Treatments
- Polyomavirus and related diseases
- Neuroblastoma Research and Treatments
- RNA Interference and Gene Delivery
- Tryptophan and brain disorders
- Lymphoma Diagnosis and Treatment
- Viral Infectious Diseases and Gene Expression in Insects
- Immune cells in cancer
- Blood groups and transfusion
- Organ Transplantation Techniques and Outcomes
- Cancer Immunotherapy and Biomarkers
- Microfluidic and Bio-sensing Technologies
- Transplantation: Methods and Outcomes
- Glioma Diagnosis and Treatment
- Childhood Cancer Survivors' Quality of Life
Bambino Gesù Children's Hospital
2015-2024
Istituti di Ricovero e Cura a Carattere Scientifico
2013-2024
Cell and Gene Therapy Catapult
2023
Gene Therapy Laboratory
2021-2022
Boston Children's Hospital
2014
Immunotherapy with chimeric antigen receptor (CAR)-expressing T cells that target the disialoganglioside GD2 expressed on tumor may be a therapeutic option for patients high-risk neuroblastoma.In an academic, phase 1-2 clinical trial, we enrolled (1 to 25 years of age) relapsed or refractory, neuroblastoma in order test autologous, third-generation GD2-CAR expressing inducible caspase 9 suicide gene (GD2-CART01).A total 27 children heavily pretreated (12 refractory disease, 14 and 1 complete...
Microenvironmental factors contribute to the immune dysfunction characterizing acute myeloid leukemia (AML). Indoleamine 2,3-dioxygenase 1 (IDO1) is an interferon (IFN)-γ-inducible enzyme that degrades tryptophan into kynurenine, which, in turn, inhibits effector T cells and promotes regulatory T-cell (Treg) differentiation. It presently unknown whether childhood AML express IDO1 activity correlates with patient outcome. We investigated expression function 37 children newly diagnosed other...
Summary Achieving complete remission ( CR ) in childhood relapsed/refractory acute lymphoblastic leukaemia ALL is a difficult task. Bortezomib, proteasome inhibitor, has vitro activity against blasts. A phase I‐ II trial, reported by the Therapeutic Advances Childhood Leukaemia and Lymphoma TACL consortium, demonstrated that bortezomib with chemotherapy acceptable toxicity remarkable patients relapsed failing 2–3 previous regimens. We evaluated combination 30 7 children B‐cell precursor BCP...
Several nonmalignant disorders (NMDs), either inherited or acquired, can be cured by allogeneic hematopoietic stem cell transplantation (HSCT). Between January 2012 and April 2020, 70 consecutive children affected primary immunodeficiencies, inherited/acquired bone marrow failure syndromes, red blood disorders, metabolic diseases, lacking a fully matched donor requiring urgent underwent TCRαβ/CD19-depleted haploidentical HSCT from an HLA-partially relative as part of prospective study. The...
Medulloblastoma (MB) is the most common malignant brain tumor in children. Despite therapeutic advancements, high-risk groups still present significant mortality. A deeper knowledge of signaling pathways contributing to MB formation and aggressiveness would help develop new successful therapies. The target rapamycin, mTOR signaling, known be involved already targetable clinical setting. Furthermore, a master metabolic regulator able control cell growth versus autophagy decisions conditions...
Abstract We report on the outcome of 24 patients with Fanconi anemia (FA) lacking an HLA matched related or unrelated donor, given HLA-haploidentical T-cell receptor αβ (TCRαβ+) and CD19+ cell-depleted hematopoietic stem cell transplantation (HSCT) in context a prospective, single-center phase 2 trial. Sustained primary engraftment was achieved 22 (91.6%) patients, median time to neutrophil recovery 12 days (range, 9-15 days) platelet 10 7-14 days). Cumulative incidences grade 1 acute...
HLA-haploidentical hematopoietic stem cell transplantation (HSCT) is suitable for patients lacking related or unrelated HLA-matched donors. Herein, we investigated whether plerixafor (MZ), as an adjunct to G-CSF, facilitated the collection of mega-doses cells (HSC) TCR-αβ/CD19-depleted haploidentical HSCT, and how this agent affects cellular graft composition. Ninety healthy donors were evaluated. Single-dose MZ was given 30 'poor mobilizers' (PM) failing attain ≥40 CD34+ HSCs/μL after 4...
TcRαβ/CD19-cell depleted HLA-haploidentical hematopoietic stem cell transplantation (haplo-HSCT) represents a new attractive platform for children affected by acute leukemia in need of an allograft and lacking matched donor, disease recurrence being the main cause treatment failure. The use zoledronic acid to enhance TcRγδ lymphocyte function after haplo-HSCT was tested open-label, feasibility, proof-of-principle study. Forty-six high-risk underwent removal TcRαβ+ CD19+ B lymphocytes. No...
Blinatumomab has remarkable efficacy in patients with relapsed/refractory (r/r) or measurable residual disease (MRD)-positive B-cell acute lymphoblastic leukemia (B-ALL). In many patients, blinatumomab treatment is followed by allogeneic hematopoietic stem cell transplantation (HSCT). However, the influence of on HSCT outcomes children and young adults (YA) remains to be fully elucidated. We conducted a single-center, retrospective analysis given as last before HSCT. Seventy-eight pediatric...
Chimeric antigen receptor T-cells (CAR T-cells) for the treatment of relapsing/refractory B-cell precursor acute lymphoblastic leukemia have led to exciting clinical results. However, CAR T-cell approaches revealed a potential risk CD19-/CAR+ leukemic relapse due inadvertent transduction cells. Background Methods We evaluated impact high percentage blast contamination in patient-derived starting material (SM) on drug product (DP) manufacturing. In vitro as well vivo models were employed...
Several diseases are associated with alterations of the B-cell compartment. Knowing how to correctly identify by flow cytometry distribution populations in peripheral blood is important help early diagnosis. In accompanying article we describe different subsets healthy donors. Here show a few examples that cause dysregulation