Publications by authors named "Sarah Vaiselbuh"

DNMT1 (DNA-methyltransferase 1) is an enzyme which contributes to the process of normal embryonic development, and aberrant expression of DNMT1 leads to tumor/leukemia progression by inducing significant changes in DNA methylation and epigenetics. We found that DNMT1 mRNA transcript is elevated in Exo-PALL compared to Exo-HD. We also confirmed and showed heightened levels of DNMT1 mRNA transcript in Exo-CM of leukemia cell lines.

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CAR-T cell therapy is not without some clinical adverse effects, namely cytokine storms, due to a massive release of cytokines when CAR-T cells multiply in the body. Our goal was to develop exosomes expressing CD19 CAR to treat CD19-positive B-cell malignancies, instead of using whole CD19 CAR-T cells, thereby reducing the clinical risk of uncontrolled cytokine storms. Exosomes are extracellular nanovesicles (30-150 nm), composed of lipids, proteins, and nucleic acids, that carry the fingerprint of their parent cells.

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We explored the effect of vincristine and prednisone on cellular and exosomal miR-181a expression in first time diagnosed leukemia and relapsed leukemia. Vincristine and prednisone induced apoptosis/pro-apoptotic genes in first time diagnosed leukemia, and suppressed the cellular and exosomal miR-181a expression. In contrast, vincristine and prednisone could not induce apoptosis/pro-apoptotic genes in relapsed leukemia, and could not change the expression of cellular or exosomal miR-181a.

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Exosomes are cell-generated nano-vesicles found in most biological fluids. Major components of their cargo are lipids, proteins, RNA, DNA, and non-coding RNAs. The miRNAs carried within exosomes reveal real-time information regarding disease status in leukemia and other cancers, and therefore exosomes have been studied as novel biomarkers for cancer.

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Exosomes are cell derived lipid nanoparticle with a size of 30-100 nm in diameter, found in almost all biological fluids. The composition of the exosomes is mainly lipid, proteins, RNA, DNA, and non-coding RNAs. Currently, most available methods and commercial kits for exosomal-RNA (Exo-RNA) isolation have limitations and shortcomings.

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Background: Langerhans cell histiocytosis (LCH) is a clonal neoplasm characterized by widely varied clinical presentations, including multisystem involvement and systemic inflammatory symptoms. The AKT pathway is relevant to survival and proliferation of dendritic cells, and is also often upregulated in hematopoietic malignancies. A clinical response in an adult patient with LCH participating in the first-in-human trial of afuresertib prompted this prospective trial.

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The majority of emergency department (ED) visits and hospitalizations for patients with sickle cell disease (SCD) are pain related. Adequate and timely pain management may improve quality of life and prevent worsening morbidities. We conducted a retrospective chart review of pediatric patients with SCD seen in the ED, selected by sickle cell-related ICD-9 codes.

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Hemophagocytic lymphohistioytosis (HLH) is a severe, life-threatening hyperinflammatory disorder that requires prompt diagnosis and treatment. Approximately, 25-50% of patients with HLH fail to achieve remission with established regimens that include dexamethasone and etoposide, or methylprednisolone and antithymocyte globulin (ATG). Some of these patients may require salvage or alternative therapeutic approaches.

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Shwachman-Diamond syndrome (SDS) is an autosomal recessive bone marrow failure syndrome typically characterized by neutropenia and pancreatic dysfunction, although phenotypic presentations vary, and the endocrine phenotype is not well-described. We report a unique case of a patient with SDS who initially presented with hypoglycemia and micropenis in the newborn period and was diagnosed with congenital hypopituitarism. We are not aware of any other cases of SDS documented with this combination of complex endocrinopathies.

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Background: Effective networking and mentorship are critical determinants of career satisfaction and success in academic medicine. The American Society of Pediatric Hematology/Oncology (ASPHO) mentoring program was developed to support Early Career (EC) members. Herein, the authors report on the initial 2-year outcomes of this novel program.

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Histiocytic disorders are rare entities that are becoming more recognized as our understanding of the molecular pathogenesis lead to novel diagnostic tests and targeted drug development. A symposium held at the American Society of Pediatric Hematology/Oncology (ASPHO) 2013 Annual Meeting discussed new insights into histiocytic disorders. This review highlights the symposium presentations, divided into three sections encompassing Langerhans cell histiocytosis (LCH), hemophagocytic lymphohistiocytosis (HLH) and Rosai Dorfman disease (RDD) including subsections on pathogenesis, clinical diagnostic criteria and novel insights into treatment.

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Background: Diversity is necessary for the survival and success of both biological and social systems including societies. There is a lack of diversity, particularly the proportion of women and minorities in leadership positions, within medicine [Leadley. AAMC 2009.

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Human mesenchymal stem cells form the supportive structure in which the functional cells of a differentiated tissue reside. We describe the creation of ectopic niches within polyurethane scaffolds coated with human mesenchymal stem cells. When implanted subcutaneously in NOD/SCID mice, these niches supported engraftment of primary human acute myeloid leukemia cells.

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