Carolyn Lutzko
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View article: Congenital dyserythropoietic anemia type I: Updated report from the congenital dyserythropoietic anemia registry of North America
Congenital dyserythropoietic anemia type I: Updated report from the congenital dyserythropoietic anemia registry of North America Open
Background Congenital dyserythropoietic anemias (CDA) are rare genetic disorders characterized by ineffective erythropoiesis and iron overload. The CDA Registry (CDAR) of North America aims to study the natural history and molecular pathog…
View article: Outcomes with Third Party Virus Specific T-cells After the Use of Single Antigen Cell Lines to Predict HLA Restriction
Outcomes with Third Party Virus Specific T-cells After the Use of Single Antigen Cell Lines to Predict HLA Restriction Open
Patients with significant T-cell dysfunction from chemotherapy or hematopoietic stem cell transplant are at significant risk for complications of viral infections. Off-the-shelf third-party virus specific T-cells (TP VSTs) are an effective…
View article: G-CSF resistance of ELANE-mutant neutropenia depends on SERF1-containing truncated–neutrophil elastase aggregates
G-CSF resistance of ELANE-mutant neutropenia depends on SERF1-containing truncated–neutrophil elastase aggregates Open
Severe congenital neutropenia (SCN) is frequently associated with dominant point mutations in ELANE, the gene encoding neutrophil elastase (NE). Chronic administration of granulocyte colony-stimulating factor (G-CSF) is a first-line treatm…
View article: A toxicology study of Csf2ra complementation and pulmonary macrophage transplantation therapy of hereditary PAP in mice
A toxicology study of Csf2ra complementation and pulmonary macrophage transplantation therapy of hereditary PAP in mice Open
Pulmonary macrophage transplantation (PMT) is a gene and cell transplantation approach in development as therapy for hereditary pulmonary alveolar proteinosis (hPAP), a surfactant accumulation disorder caused by mutations in CSF2RA/B
View article: Third-Party and Patient-Specific Donor-Derived Virus-Specific T Cells Demonstrate Similar Efficacy and Safety for Management of Viral Infections after Hematopoietic Stem Cell Transplantation in Children and Young Adults
Third-Party and Patient-Specific Donor-Derived Virus-Specific T Cells Demonstrate Similar Efficacy and Safety for Management of Viral Infections after Hematopoietic Stem Cell Transplantation in Children and Young Adults Open
Infections with double-stranded DNA viruses are a common complication after hematopoietic stem cell transplantation (HSCT) and cause significant morbidity and mortality in the post-transplantation period. Both donor-derived (DD) and third-…
View article: P1453: STABLE TRANSDUCTION OF FETAL HEMOGLOBIN IN PATIENTS WITH SICKLE CELL DISEASE IN THE PHASE 1/2 MOMENTUM STUDY OF ARU-1801 GENE THERAPY AND REDUCED INTENSITY CONDITIONING
P1453: STABLE TRANSDUCTION OF FETAL HEMOGLOBIN IN PATIENTS WITH SICKLE CELL DISEASE IN THE PHASE 1/2 MOMENTUM STUDY OF ARU-1801 GENE THERAPY AND REDUCED INTENSITY CONDITIONING Open
Background: Sickle cell disease (SCD) is a genetic red blood cell disorder that causes chronic hemolytic anemia, progressive organ damage, and painful vaso-occlusive crises. ARU-1801 is a gene therapy for SCD, designed to produce HbFG16D e…
View article: Scheduled administration of virus-specific T cells for viral prophylaxis after pediatric allogeneic stem cell transplant
Scheduled administration of virus-specific T cells for viral prophylaxis after pediatric allogeneic stem cell transplant Open
Infections with double-stranded DNA viruses are a significant cause of morbidity and mortality in pediatric patients following allogeneic hematopoietic stem cell transplantation (HSCT). Virus-specific T-cell therapies (VSTs) have been show…
View article: Virus-specific T cells for adenovirus infection after stem cell transplantation are highly effective and class II HLA restricted
Virus-specific T cells for adenovirus infection after stem cell transplantation are highly effective and class II HLA restricted Open
Infection with adenoviruses is a common and significant complication in pediatric patients after allogeneic hematopoietic stem cell transplantation. Treatment options with traditional antivirals are limited by poor efficacy and significant…
View article: Complementation of <i>CSF2RA</i> Mutations Restores GM-CSF Signaling to Macrophages from Patients with Hereditary Pulmonary Alveolar Proteinosis
Complementation of <i>CSF2RA</i> Mutations Restores GM-CSF Signaling to Macrophages from Patients with Hereditary Pulmonary Alveolar Proteinosis Open
View article: Congenital dyserythropoietic anemia type I: First report from the Congenital Dyserythropoietic Anemia Registry of North America (CDAR)
Congenital dyserythropoietic anemia type I: First report from the Congenital Dyserythropoietic Anemia Registry of North America (CDAR) Open
View article: Virus-specific T-cell therapy to treat BK polyomavirus infection in bone marrow and solid organ transplant recipients.
Virus-specific T-cell therapy to treat BK polyomavirus infection in bone marrow and solid organ transplant recipients. Open
BK polyomavirus (BKPyV) infection is a major complication of hematopoietic stem cell transplant (HSCT) and solid organ transplant (SOT). Treatment options are limited, poorly effective, and have significant toxicities. Cellular therapy usi…
View article: VPS4A Mutations in Humans Cause Syndromic Congenital Dyserythropoietic Anemia due to Cytokinesis and Trafficking Defects
VPS4A Mutations in Humans Cause Syndromic Congenital Dyserythropoietic Anemia due to Cytokinesis and Trafficking Defects Open
View article: Complement inhibition does not impair the clinical antiviral capabilities of virus-specific T-cell therapy
Complement inhibition does not impair the clinical antiviral capabilities of virus-specific T-cell therapy Open
Key Points The use of terminal complement blockade is compatible with virus-specific T-cell (VST) expansion and clinical effectiveness. VST and complement-blocking agent concurrent therapy may be safely used in patients with thrombotic mic…
View article: Mouse models of neutropenia reveal progenitor-stage-specific defects
Mouse models of neutropenia reveal progenitor-stage-specific defects Open
View article: Characterization of Viral Epitopes and the HLA Restriction That Govern Anti-Adenoviral Response to Viral Specific T-Lymphocyte Therapy in a Pediatric Cohort
Characterization of Viral Epitopes and the HLA Restriction That Govern Anti-Adenoviral Response to Viral Specific T-Lymphocyte Therapy in a Pediatric Cohort Open
IntroductionAdenoviral (AdV) infections are common after HSCT and are a major cause of complications. Antiviral medications are frequently ineffective and toxic. At CCHMC, viral specific T cells (VST) from 3rd party donors are generated by…
View article: The Natural History of BK Polyomavirus and the Host Immune Response After Stem Cell Transplantation
The Natural History of BK Polyomavirus and the Host Immune Response After Stem Cell Transplantation Open
Background BK polyomavirus (BKPyV) is associated with symptomatic hemorrhagic cystitis after hematopoietic cell transplantation (HCT). Little is known about the host immune response, effectiveness of antiviral treatment, or impact of asymp…
View article: CD38brightCD8+ T Cells Associated with the Development of Acute GVHD Are Activated, Proliferating, and Cytotoxic Trafficking Cells
CD38brightCD8+ T Cells Associated with the Development of Acute GVHD Are Activated, Proliferating, and Cytotoxic Trafficking Cells Open
View article: Neutrophils Derived from Genetically Modified Human Induced Pluripotent Stem Cells Circulate and Phagocytose Bacteria In Vivo
Neutrophils Derived from Genetically Modified Human Induced Pluripotent Stem Cells Circulate and Phagocytose Bacteria In Vivo Open
Bacterial and fungal infections are a major cause of morbidity and mortality in neutropenic patients. Donor-derived neutrophil transfusions have been used for prophylaxis or treatment for infection in neutropenic patients. However, the sho…
View article: Building a Third-Party VST Bank From Scratch—the Cincinnati Experience
Building a Third-Party VST Bank From Scratch—the Cincinnati Experience Open
Background: Viral infections remain a challenge to treat post HCT in children, and significantly contribute to morbidity and mortality. Virus specific T cells (VSTs) have shown tremendous clinical efficacy in treating viral infections post…
View article: Production of Trivalent Antigen-Specific T Cells Utilizing Viral Peptide Stimulation
Production of Trivalent Antigen-Specific T Cells Utilizing Viral Peptide Stimulation Open
Virus-specific T cells (VSTs) have shown promising results in several clinical trials, utilizing effector cells that were isolated and expanded in one of three different ways. Our center has recently converted from utilizing transduced EBV…
View article: Molecular, phenotypic, and sample-associated data to describe pluripotent stem cell lines and derivatives
Molecular, phenotypic, and sample-associated data to describe pluripotent stem cell lines and derivatives Open
View article: Integrated Genomic Analysis of Diverse Induced Pluripotent Stem Cells from the Progenitor Cell Biology Consortium
Integrated Genomic Analysis of Diverse Induced Pluripotent Stem Cells from the Progenitor Cell Biology Consortium Open
The rigorous characterization of distinct induced pluripotent stem cells (iPSC) derived from multiple reprogramming technologies, somatic sources, and donors is required to understand potential sources of variability and downstream potenti…
View article: 444. Large Scale Culture and Differentiation of Induced Pluripotent Stem Cells for Neutrophil Replacement Therapies
444. Large Scale Culture and Differentiation of Induced Pluripotent Stem Cells for Neutrophil Replacement Therapies Open
Neutrophils are a key component in the innate immune system and are crucial in the protection against bacterial and fungal infections. Patients with neutropenias are at high risk to develop serious life threatening bacterial and fungal inf…
View article: Production and purification of high-titer foamy virus vector for the treatment of leukocyte adhesion deficiency
Production and purification of high-titer foamy virus vector for the treatment of leukocyte adhesion deficiency Open
Compared to other integrating viral vectors, foamy virus (FV) vectors have distinct advantages as a gene transfer tool, including their nonpathogenicity, the ability to carry larger transgene cassettes, and increased stability of virus par…
View article: Pathogenesis of ELANE-mutant severe neutropenia revealed by induced pluripotent stem cells
Pathogenesis of ELANE-mutant severe neutropenia revealed by induced pluripotent stem cells Open
Severe congenital neutropenia (SCN) is often associated with inherited heterozygous point mutations in ELANE, which encodes neutrophil elastase (NE). However, a lack of appropriate models to recapitulate SCN has substantially hampered the …
View article: 12. GM-CSF But Not M-CSF is Critical to the Therapeutic Mechanism of Action and Efficacy of Pulmonary Macrophage Transplantation (PMT) Therapy of Hereditary Pulmonary Alveolar Proteinosis (hPAP)
12. GM-CSF But Not M-CSF is Critical to the Therapeutic Mechanism of Action and Efficacy of Pulmonary Macrophage Transplantation (PMT) Therapy of Hereditary Pulmonary Alveolar Proteinosis (hPAP) Open
Rationale: Hereditary pulmonary alveolar proteinosis (hPAP) is a disorder caused by recessive mutations in the GM-CSF receptor α or β gene (CSF2RA or CSF2RB), which is characterized by surfactant accumulation and hypoxemic respiratory fail…
View article: 461. Scale-Up and Manufacturing of High-Titer Foamy Virus Vector Containing Human CD18 for the Treatment of Leukocyte Adhesion Deficiency
461. Scale-Up and Manufacturing of High-Titer Foamy Virus Vector Containing Human CD18 for the Treatment of Leukocyte Adhesion Deficiency Open
Compared to other integrating viral vectors, foamy virus (FV) vectors have several distinct advantages as a gene transfer tool, including: the ability to carry larger expression cassettes, the increased stability due to a DNA genome formed…