A5051468151- Lysosomal Storage Disorders Research
- Hematopoietic Stem Cell Transplantation
- Peroxisome Proliferator-Activated Receptors
- Metabolism and Genetic Disorders
- Glycogen Storage Diseases and Myoclonus
- Acute Lymphoblastic Leukemia research
- Neurogenetic and Muscular Disorders Research
- Acute Myeloid Leukemia Research
- Bone Metabolism and Diseases
- Virus-based gene therapy research
- Biomedical Research and Pathophysiology
- Neuroinflammation and Neurodegeneration Mechanisms
- Cytomegalovirus and herpesvirus research
- Bone health and treatments
- Trypanosoma species research and implications
- Neonatal Health and Biochemistry
- Cancer, Hypoxia, and Metabolism
- CAR-T cell therapy research
- Immune cells in cancer
- Immune Cell Function and Interaction
- Chronic Lymphocytic Leukemia Research
- Eicosanoids and Hypertension Pharmacology
- RNA regulation and disease
- Autoimmune and Inflammatory Disorders Research
- Blood disorders and treatments
University of Minnesota
2016-2025
Fairview Health Services
1996-2025
University College London
2022-2024
University of Minnesota System
2000-2024
Inserm
2004-2024
University of Minnesota Medical Center
2005-2024
Cancer Research Center
2024
Dana-Farber/Boston Children's Cancer and Blood Disorders Center
2024
Harvard University
2024
Boston Children's Hospital
2024
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...
Approximately 20,000 hematopoietic cell transplantation (HCT) procedures are performed in the United States annually. With advances technology and supportive care practices, HCT has become safer, patient survival continues to improve over time. Indications for continue evolve as research refines role established indications identifies emerging where may be beneficial. The American Society Blood Marrow Transplantation (ASBMT) a multiple-stakeholder task force consisting of transplant experts,...
Gene therapy with elivaldogene autotemcel (eli-cel) consisting of autologous CD34+ cells transduced lentiviral vector containing
This cohort study conducted among Minnesota children diagnosed with adrenoleukodystrophy through newborn screening examines correlation of C26:0 lysophosphatidylcholine (C26LPC) clinical phenotype over 5 years and recommends adjusting early childhood surveillance regimens in lower C26LPC levels.
Among 94 osteopetrotic patients presenting with a severe clinical picture and diagnosed early in life, 12 bore mutations the ClCN7 gene, but only 7 of them had expected two recessive mutations. The remaining five seem to be heterozygous for mutation, significant variations were observed manifestations their disease, even within same family.Human osteopetroses are heterogeneous group diseases that include both infantile severe, autosomal (ARO) adult dominant (ADO) forms. Two genes, Atp6a3...
Autologous hematopoietic stem cell transplantation (ASCT) has become standard therapy for primary refractory (PR REF) or relapsed (REL) Hodgkin's lymphoma (HL); however, more than half of these patients eventually relapse and die their disease. We studied long-term outcomes evaluated factors influencing progression-free survival (PFS) in 141 with PR REF REL HL who underwent ASCT between 1985 2003. Median age at was 30 years (range, 7–60 years); 21 had REF, 120 HL. With a median follow-up 6.3...
Metachromatic Leukodystrophy (MLD) is a rare, fatal demyelinating disorder with limited treatment options. Published outcomes after hematopoietic stem cell transplantation (HSCT) are scant and mixed. We report survival function following HSCT for large, single-center MLD cohort.Transplant-related data, serial measures (brain MRI, nerve conduction velocity (NCV), neurologic neuropsychology evaluations) were reviewed. When possible, parental interviews informed current status, quality-of-life,...
Cerebrospinal fluid (CSF) and serum levels of 12 cytokines or chemokines important in central nervous system (CNS) infections were measured 76 Ugandan children with cerebral malaria (CM) 8 control children. As compared children, had higher cerebrospinal interleukin (IL)-6, CXCL-8/IL-8, granulocyte-colony stimulating factor (G-CSF), tumor necrosis factor-alpha (TNF-alpha), IL-1 receptor antagonist. There was no correlation between cytokine for any except G-CSF. Elevated but not TNF-alpha on...
Abstract Tissue organoids are a promising technology that may accelerate development of the societal and NIH mandate for precision medicine. Here we describe robust simple method generating cerebral (cOrgs) from human pluripotent stem cells by using chemically defined hydrogel material culture medium. By no additional neural induction components, cOrgs appeared on surface within 10–14 days, under static conditions, they attained sizes up to 3 mm in greatest dimension day 28. Histologically,...
Cerebral adrenoleukodystrophy (CALD) is a rapidly progressing, often fatal neurodegenerative disease caused by mutations in the ABCD1 gene, resulting deficiency of ALD protein. Clinical benefit has been reported following allogeneic hematopoietic stem cell transplantation (HSCT). We conducted large multicenter retrospective chart review to characterize natural history CALD, describe outcomes after HSCT, and identify predictors treatment outcomes. Major functional disabilities (MFDs) were...
Central nervous system Langerhans cell histiocytosis (CNS-LCH) brain involvement may include mass lesions and/or a neurodegenerative disease (LCH-ND) of unknown etiology. The goal this study was to define the mechanisms pathogenesis that drive CNS-LCH.Cerebrospinal fluid (CSF) biomarkers including CSF proteins and extracellular BRAFV600E DNA were analyzed in from patients with CNS-LCH compared tumors other conditions. Additionally, presence tested peripheral mononuclear blood cells (PBMCs)...