Mol Ther Methods Clin Dev
December 2024
Mucopolysaccharidosis type I (MPS I) is a metabolic disorder characterized by a deficiency in α-l-iduronidase (IDUA), leading to impaired glycosaminoglycan degradation. Current approved treatments seek to restore IDUA levels via enzyme replacement therapy (ERT) and/or hematopoietic stem cell transplantation (HSCT). The effectiveness of these treatment strategies in preventing neurodegeneration is limited due to the inability of ERT to penetrate the blood-brain barrier (BBB) and HSCT's limited CNS reconstitution of IDUA levels.
View Article and Find Full Text PDFHemorrhagic shock and subsequent resuscitation can cause significant dysregulation of critical systems, including the vascular endothelium. Following hemorrhage, the endothelial lining (glycocalyx) can shed, causing release of glycocalyx components, endothelial activation, and systemic inflammation. A canine model of hemorrhagic shock was used to evaluate five resuscitation fluids, including Lactated Ringers+Hetastarch, Whole Blood (WB), Fresh Frozen Plasma+packed Red Blood Cells (FFP+pRBC), and two hemoglobin-based oxygen carrier (HBOC) fluids, for their impact on glycocalyx shedding.
View Article and Find Full Text PDFBackground: Sickle cell disease (SCD) exemplifies many of the social, racial, and healthcare equity issues in the United States. Despite its high morbidity, mortality, and cost of care, SCD has not been prioritized in research and clinical teaching, resulting in under-trained clinicians and a poor evidence base for managing complications of the disease. This study aimed to perform a needs assessment, examining the perspectives of medical trainees pursuing hematology/oncology subspecialty training regarding SCD-focused education and clinical care.
View Article and Find Full Text PDFRespiratory syncytial virus (RSV) primarily affects infants and children and can manifest as upper airway dysfunction. Patients at the highest risk of increased morbidity and mortality from RSV include those who are immunosuppressed and the elderly. Patients with RSV hepatitis most commonly present with fever, abdominal pain, nausea, and vomiting; however, patients may present with jaundice and coagulopathies in a severe infection.
View Article and Find Full Text PDFSubst Abuse Treat Prev Policy
April 2024
We aimed to learn the experiences of clinicians and adolescents and young adults with sickle cell disease (AYA-SCD) with managing their disease at home and making medical decisions as they transition from pediatric to adult care, and their perceptions of a video game intervention to positively impact these skills. We conducted individual, semistructured interviews with patients (AYA-SCD ages 15 to 26 years) and clinicians who provide care to AYA-SCD at an urban, quaternary-care hospital. Interviews elicited patients' and clinicians' experiences with AYA-SCD, barriers and facilitators to successful home management, and their perspectives on shared decision-making and a video game intervention.
View Article and Find Full Text PDFIEEE Trans Med Imaging
May 2024
Color fundus photography (CFP) and Optical coherence tomography (OCT) images are two of the most widely used modalities in the clinical diagnosis and management of retinal diseases. Despite the widespread use of multimodal imaging in clinical practice, few methods for automated diagnosis of eye diseases utilize correlated and complementary information from multiple modalities effectively. This paper explores how to leverage the information from CFP and OCT images to improve the automated diagnosis of retinal diseases.
View Article and Find Full Text PDFObjective: To assess the feasibility of daily Home OCT imaging among patients with neovascular age-related macular degeneration (nAMD).
Design: Prospective observational study.
Participants: Participants with ≥ 1 eye with previously untreated nAMD and visual acuity 20/20 to 20/320.
We report in this study on the isolation and expansion of neural crest stem cells (NCSCs) from the epithelium of oral mucosa (OM) using reagents that are GMP-certified and FDA-approved for clinical use. Characterization analysis showed that the levels of keratins , , , , , and -specific to OM epithelial cells-were significantly lower in the experimental NCSCs. While was decreased with no statistically significant difference, the earliest neural crest specifier genes , , , , , , and showed a trend in increased expression in NCSCs.
View Article and Find Full Text PDFBackground: Gene therapy is a potential cure for sickle cell disease (SCD). Conventional cost-effectiveness analysis (CEA) does not capture the effects of treatments on disparities in SCD, but distributional CEA (DCEA) uses equity weights to incorporate these considerations.
Objective: To compare gene therapy versus standard of care (SOC) in patients with SCD by using conventional CEA and DCEA.
Importance: Pain related to sickle cell disease (SCD) is complex and associated with social determinants of health. Emotional and stress-related effects of SCD impact daily quality of life and the frequency and severity of pain.
Objective: To explore the association of educational attainment, employment status, and mental health with pain episode frequency and severity among individuals with SCD.
Obesity results from the expansion of adipose tissue, a versatile tissue regulating energy homeostasis, adipokine secretion, thermogenesis, and inflammation. The primary function of adipocytes is thought to be lipid storage through lipid synthesis, which is presumably intertwined with adipogenesis. However, during prolonged fasting, adipocytes are depleted of lipid droplets yet retain endocrine function and an instant response to nutrients.
View Article and Find Full Text PDFThe ability to anticipate and process predictable unpleasant events, while also regulating emotional reactivity, is an adaptive skill. The current article and a companion in this issue test for potential changes in predictable event processing across the childhood-to-adolescence transition, a key developmental period for biological systems that support cognitive/emotional abilities. While the companion article focuses on emotion regulation and peripheral attention modulation in predictable unpleasant contexts, the current paper presents neurophysiological markers of predictable event processing itself.
View Article and Find Full Text PDFThe ability to anticipate and process predictable unpleasant events, while also regulating emotional reactivity, is an adaptive skill. The current article and a companion in this issue test for potential changes in predictable event processing across the childhood-to-adolescence transition, a key developmental period for biological systems that support cognitive/ emotional abilities. While the companion article focuses on neurophysiology of predictable event processing itself, the present article examines peripheral emotional response regulation and attention modulation that coincides with event processing.
View Article and Find Full Text PDFThe American Society of Hematology Clinical Research Training Institute (CRTI) is a clinical research training program with a competitive application process. The objectives were to compare application scores based on applicant and reviewer sex and underrepresented minority (URM) status. We included applications to CRTI from 2003 to 2019.
View Article and Find Full Text PDFIntroduction: Sickle cell disease (SCD) is a chronic condition with progressive neurocognitive deficits. Health literacy (HL) is essential during adolescence and young adulthood, as the transition to adult care requires healthcare decisions. HL is known to be low in SCD; however, relation between general cognitive ability and HL has not been investigated.
View Article and Find Full Text PDFBackground: Health sciences librarians and nursing journal contributors have expressed concern about the impact of using strict parameters when searching the literature.
Purpose: The purpose of this study was to explore the use of strict search criteria (eg, 5-year rule, "nurse as author") by direct care nurses and nursing students.
Methods: Fourteen online focus groups were conducted with 54 participants: direct care nurses, health sciences librarians, nursing faculty, and nursing students.
As a surging public health crisis, obesity and overweight predispose individuals to various severe comorbidities contributed by the accompanying chronic inflammation. However, few options exist for tackling chronic inflammation in obesity or inhibiting depot-specific adiposity. Here, we report that polycationic polyamidoamine (PAMAM) treatment can improve both aspects of obesity.
View Article and Find Full Text PDFBackground: Music is part of operating room (OR) culture; however, some personnel may perceive music as a distraction.
Methods: A single institution survey of surgeons (SURG), anesthesia (ANES), and nursing (NURS) regarding attitudes on music in the OR.
Results: There were 222 responses (67% response rate) agreeing that music in the OR should be allowed (91%), is calming (75%), and helps with focus (63%).
Depression and anxiety disorders overlap in clinical populations, suggesting common mechanisms that may be further investigated in reliable animal models. We used filial 8 female Long-Evans rats bred for high (HAn; n = 19) and low anxiety (LAn)-like behavior (n = 21) to assess forced swim test mobility strategies and chronic mild stress (CMS)-induced depression-like symptoms. We measured (1) weight, (2) fur piloerection, (3) sweet food consumption, (4) grooming behavior, and (5) circulating estradiol (E2).
View Article and Find Full Text PDFThis theoretical review proposes an integrated biopsychosocial model for stress recovery, highlighting the interconnectedness of intra- and interpersonal coping processes. The proposed model is conceptually derived from prior research examining interpersonal dynamics in the context of stressor-related disorders, and it highlights interconnections between relational partner dynamics, perceived self-efficacy, self-discovery, and biological stress responsivity during posttraumatic recovery. Intra- and interpersonal processes are discussed in the context of pre-, peri-, and post-trauma stress vulnerability as ongoing transactions occurring within the individual and between the individual and their environment.
View Article and Find Full Text PDFBackground: Sickle cell disease (SCD), an inherited red blood cell disorder, primarily affects African Americans in the United States. Adolescents and young adults with SCD (AYA-SCD) are at risk of high morbidity and mortality when transitioning from pediatric to adult care. The goal of this qualitative study was to understand factors associated with optimal implementation of the AYA-SCD transition.
View Article and Find Full Text PDFHeart disease is a leading cause of death for African Americans. A community-academic partnership cross-trained community health workers to engage African American adults in a 6-month heart health education and risk reduction intervention. We conducted a one-group feasibility study using a one group (pre-posttest) design.
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