Satiro De Oliveira
A5031879484- CAR-T cell therapy research
- Virus-based gene therapy research
- Hematopoietic Stem Cell Transplantation
- Acute Lymphoblastic Leukemia research
- CRISPR and Genetic Engineering
- Peroxisome Proliferator-Activated Receptors
- RNA Interference and Gene Delivery
- Immunodeficiency and Autoimmune Disorders
- Immune Cell Function and Interaction
- Polyomavirus and related diseases
- Acute Myeloid Leukemia Research
- Neuroblastoma Research and Treatments
- Hemoglobinopathies and Related Disorders
- Childhood Cancer Survivors' Quality of Life
- Cytomegalovirus and herpesvirus research
- Metabolism and Genetic Disorders
- Chronic Myeloid Leukemia Treatments
- T-cell and B-cell Immunology
- Eicosanoids and Hypertension Pharmacology
- Blood disorders and treatments
- Prenatal Screening and Diagnostics
- Renal and related cancers
- Cancer-related molecular mechanisms research
- Parvovirus B19 Infection Studies
- Circular RNAs in diseases
University of California, Los Angeles
2016-2025
Mattel Children's Hospital
2014-2025
University College London
2024
Health Net
2024
Massachusetts General Hospital
2024
Dana-Farber/Boston Children's Cancer and Blood Disorders Center
2024
Harvard University
2024
Inserm
2024
Université Paris-Saclay
2024
UCLA Jonsson Comprehensive Cancer Center
2023
In X-linked adrenoleukodystrophy, mutations in ABCD1 lead to loss of function the ALD protein. Cerebral adrenoleukodystrophy is characterized by demyelination and neurodegeneration. Disease progression, which leads neurologic death, can be halted only with allogeneic hematopoietic stem-cell transplantation.We enrolled boys cerebral a single-group, open-label, phase 2-3 safety efficacy study. Patients were required have early-stage disease gadolinium enhancement on magnetic resonance imaging...
In previous clinical trials involving children with X-linked severe combined immunodeficiency (SCID-X1), a Moloney murine leukemia virus-based γ-retrovirus vector expressing interleukin-2 receptor γ-chain (γc) complementary DNA successfully restored immunity in most patients but resulted vector-induced through enhancer-mediated mutagenesis 25% of patients. We assessed the efficacy and safety self-inactivating retrovirus for treatment SCID-X1.
Severe combined immunodeficiency due to adenosine deaminase (ADA) deficiency (ADA-SCID) is a rare and life-threatening primary immunodeficiency.
Cerebral adrenoleukodystrophy is a severe form of X-linked characterized by white-matter disease, loss neurologic function, and early death. Elivaldogene autotemcel (eli-cel) gene therapy, which consists autologous CD34+ cells transduced with Lenti-D lentiviral vector containing
X-linked hyper-immunoglobulin M (hyper-IgM) syndrome (XHIM) is a primary immunodeficiency due to mutations in CD40 ligand that affect immunoglobulin class-switch recombination and somatic hypermutation. The disease amenable gene therapy using retroviral vectors, but dysregulated expression results abnormal lymphoproliferation mouse models, highlighting the need for alternative strategies. Here, we demonstrate ability of both transcription activator-like effector nuclease (TALEN) clustered...
Significance Commercial strategies to deliver biomolecular cargo ex vivo (e.g., electroporation, lipofection) clinically relevant cell lines are limited by toxicity, cost, and throughput. These technical limitations have inhibited development of these technologies into streamlined clinical platforms for manufacturing gene-modified stem cells cancer immunotherapies. Here, we demonstrate an acoustofluidic platform capable delivering plasmids with high throughput human T lymphocytes, peripheral...
The β2 common integrin subunit CD18 is essential for leukocyte-endothelial adhesion and extravasation to inflamed or infected tissue. Damaging variants in ITGB2, which encodes CD18, cause leukocyte deficiency type I (LAD-I), an inborn error of immunity that leads frequent life-threatening infections a high risk death among affected children. Allogeneic hematopoietic stem-cell transplantation (HSCT) represents curative treatment but limited by donor availability, incidence graft-versus-host...
Autologous hematopoietic stem cell gene therapy is an approach to treating sickle disease (SCD) patients that may result in lower morbidity than allogeneic transplantation. We examined the potential of a lentiviral vector (LV) (CCL-βAS3-FB) encoding human hemoglobin (HBB) engineered impede polymerization (HBBAS3) transduce BM CD34+ cells from SCD donors and prevent sickling red blood produced by vitro differentiation. The CCL-βAS3-FB LV transduced either healthy or at similar levels, based...
BACKGROUND. Autologous hematopoietic stem cell transplantation (HSCT) of gene-modified cells is an alternative to enzyme replacement therapy (ERT) and allogeneic HSCT that has shown clinical benefit for adenosine deaminase–deficient (ADA-deficient) SCID when combined with reduced intensity conditioning (RIC) ERT cessation. Clinical safety therapeutic efficacy were evaluated in a phase II study.
Abstract Viral infections remain a major risk in immunocompromised pediatric patients, and virus-specific T cell (VST) therapy has been successful for treatment of refractory viral prior studies. We performed phase II multicenter study (NCT03475212) the patients with inborn errors immunity and/or post allogeneic hematopoietic stem transplant using partially-HLA matched VSTs targeting cytomegalovirus, Epstein-Barr virus, or adenovirus. Primary endpoints were feasibility, safety, clinical...
Allogeneic hematopoietic cell transplantation (HCT) can be complicated by life-threatening organ toxicity and infection necessitating intensive care. Epidemiologic data have been limited single-center studies, poor database granularity, a lack of long-term survivors. To identify contemporary trends in care unit (ICU) use outcomes, we merged from the Center for International Blood Marrow Transplant Research Virtual Pediatric Systems databases. We identified 6995 pediatric patients with HCT...
Chimeric antigen receptors (CARs) against CD19 have been shown to direct T-cells specifically target B-lineage malignant cells in animal models and clinical trials, with efficient tumor cell lysis. However, some cases, there has insufficient persistence of effector cells, limiting efficacy. We propose gene transfer hematopoietic stem/progenitor (HSPC) as a novel approach deliver the CD19-specific CAR, potential for ensuring persistent production multiple lineages targeting cells. Assessments...
Using gene modification of hematopoietic stem cells (HSC) to create persistent generation multilineage immune effectors target cancer directly is proposed. Gene-modified human HSC have been used introduce genes correct, prevent, or treat diseases. Concerns regarding malignant transformation, abnormal hematopoiesis, and autoimmunity exist, making the co-delivery a suicide necessary safety measure. Truncated epidermal growth factor receptor (EGFRt) was tested as system co-delivered with...
The use of post-transplantation cyclophosphamide (PTCy) for graft-versus-host disease (GVHD) prophylaxis in recipients haploidentical and fully matched transplantations is on the increase. Published studies have reported an increased incidence cytomegalovirus (CMV) infection with PTCy. Limited data exist outcomes non-CMV herpesviruses (NCHV) this setting. aim study was to evaluate cumulative NCHV infections association transplantation-specific transplantation PTCy (HaploCy), sibling donor...
Chimeric Antigen Receptors (CARs) consist of the antigen-recognition portion a monoclonal antibody fused to an intracellular signaling domain capable activating T-cells. CARs displayed on surface transduced cells perform non-MHC-restricted antigen recognition and pathways for induction target cytolysis, cytokine secretion proliferation. Clinical trials are in progress assessing use mature T-lymphocytes with targeting CD19 treat B-lineage malignancies. is attractive immunotherapy because its...
Cytogenetic risk stratification at diagnosis has long been one of the most useful tools to assess prognosis in acute lymphoblastic leukemia (ALL). To examine prognostic impact cytogenetic abnormalities on outcomes after allogeneic hematopoietic cell transplantation, we studied 1731 adults with Philadelphia-negative ALL complete remission who underwent myeloablative or reduced intensity/non-myeloablative conditioning transplant from unrelated matched sibling donors reported Center for...
Background: Pediatric hematopoietic stem cell transplant (pHSCT) patients are at risk for many life-threatening post-transplant complications, notably relapse, graft-versus-host disease (GvHD), and infection. Methods: This retrospective study reviewed 10 years of pHSCT a single institution, assessing factors post-transplantation viral infections (herpes simplex virus (HSV), varicella-zoster (VZV), Epstein-Barr (EBV), cytomegalovirus (CMV), human herpes 6 (HHV6), adenovirus (ADNV), polyoma 1...
The NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines) for Pediatric Acute Lymphoblastic Leukemia (ALL) were developed as a result of meetings convened by multidisciplinary panel pediatric ALL experts, with the goal providing recommendations on standard treatment approaches based current evidence. focus risk assessment and stratification risk-adapted therapy; strategies BCR::ABL1 (Philadelphia chromosome [Ph])-negative -positive B-cell lineage, T-cell infant ALL; supportive care...
Patients with refractory or recurrent B-lineage hematologic malignancies have less than 50% of chance cure despite intensive therapy and innovative approaches are needed. We hypothesize that gene modification haematopoietic stem cells (HSC) an anti-CD19 chimeric antigen receptor (CAR) will produce a multi-lineage, persistent immunotherapy against can be controlled by the HSVsr39TK suicide gene. High-titer third-generation self-inactivating lentiviral constructs were developed to deliver...
The Blood and Marrow Transplant Clinical Trials Network (BMT CTN 0501) randomized children with hematologic malignancies to transplantation 1 or 2 cord blood units (UCB) between 2006 2012. While the trial concluded that survival was similar regardless of number infused, better than previously reported. This prompted a comparison versus nontrial patients determine generalizability results whether because treatment regimen. During period, 396 recipients single UCB unit met eligibility but were...