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2019

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Articles 3571 - 3600 of 16112

Full-Text Articles in Medicine and Health Sciences

Prevalence Of Complications In Type 2 Diabetics In Dubai, Uae: A Cross-Sectional Study, Haleama Al Sabbah, Moaza Alketbi, Rania Dghaim Aug 2019

Prevalence Of Complications In Type 2 Diabetics In Dubai, Uae: A Cross-Sectional Study, Haleama Al Sabbah, Moaza Alketbi, Rania Dghaim

All Works

Background: Diabetes complications have been increasingly prevalent among type 2 diabetics during the past decades causing high rates of morbidity and mortality. Measures of the prevalence of diabetes complications will lead to preventive decisions and planning of health care. The main objective of this study was to assess the prevalence rates of complications in Type 2 diabetics in two Diabetes Centers in Dubai. Methods: A cross-sectional descriptive analytical study conducted among type 2 diabetics attending diabetes centers in Dubai. Data was collected form secondary source using patients' records from two diabetes centers involved in the study. Random sampling technique was …


A First Genetic Portrait Of Synaptonemal Complex Variation., Richard J Wang, Beth L Dumont, Peicheng Jing, Bret A Payseur Aug 2019

A First Genetic Portrait Of Synaptonemal Complex Variation., Richard J Wang, Beth L Dumont, Peicheng Jing, Bret A Payseur

Faculty Research 2019

The synaptonemal complex (SC) is a proteinaceous scaffold required for synapsis and recombination between homologous chromosomes during meiosis. Although the SC has been linked to differences in genome-wide crossover rates, the genetic basis of standing variation in SC structure remains unknown. To investigate the possibility that recombination evolves through changes to the SC, we characterized the genetic architecture of SC divergence on two evolutionary timescales. Applying a novel digital image analysis technique to spermatocyte spreads, we measured total SC length in 9,532 spermatocytes from recombinant offspring of wild-derived mouse strains with differences in this fundamental meiotic trait. Using this large …


Week Of August 26, 2019, New York Medical College Aug 2019

Implementing A Health Coach Into Your Healthcare Continuum, Dalton Jarvis Aug 2019

Implementing A Health Coach Into Your Healthcare Continuum, Dalton Jarvis

Masters Theses/Capstone Projects

Presentation on Implementing a Health-coach into your Healthcare Continuum.


Abroad View Of Wellness, Patrick Kunkel, Megan Mills, Seth Clark Aug 2019

Abroad View Of Wellness, Patrick Kunkel, Megan Mills, Seth Clark

Masters Theses/Capstone Projects

A presentation on a Study Abroad trip taken to Hungary.


Liquid Biopsy-Based Single-Cell Metabolic Phenotyping Of Lung Cancer Patients For Informative Diagnostics., Ziming Li, Zhuo Wang, Yin Tang, Xiang Lu, Jie Chen, Yu Dong, Baojun Wu, Chunying Wang, Liu Yang, Zhili Guo, Min Xue, Shun Lu, Wei Wei, Qihui Shi Aug 2019

Liquid Biopsy-Based Single-Cell Metabolic Phenotyping Of Lung Cancer Patients For Informative Diagnostics., Ziming Li, Zhuo Wang, Yin Tang, Xiang Lu, Jie Chen, Yu Dong, Baojun Wu, Chunying Wang, Liu Yang, Zhili Guo, Min Xue, Shun Lu, Wei Wei, Qihui Shi

Articles, Abstracts, and Reports

Accurate prediction of chemo- or targeted therapy responses for patients with similar driver oncogenes through a simple and least-invasive assay represents an unmet need in the clinical diagnosis of non-small cell lung cancer. Using a single-cell on-chip metabolic cytometry and fluorescent metabolic probes, we show metabolic phenotyping on the rare disseminated tumor cells in pleural effusions across a panel of 32 lung adenocarcinoma patients. Our results reveal extensive metabolic heterogeneity of tumor cells that differentially engage in glycolysis and mitochondrial oxidation. The cell number ratio of the two metabolic phenotypes is found to be predictive for patient therapy response, physiological …


Examination Of Human Health Impacts Due To Adverse Climate Events Through The Use Of Vulnerability Mapping: A Scoping Review, Michael T. Schmeltz, Peter J. Marcotullio Aug 2019

Examination Of Human Health Impacts Due To Adverse Climate Events Through The Use Of Vulnerability Mapping: A Scoping Review, Michael T. Schmeltz, Peter J. Marcotullio

Publications and Research

Government officials, health professionals, and other decision makers are tasked with characterizing vulnerability and understanding how populations experience risks associated with exposure to climate-related hazards. Spatial analyses of vulnerable locations have given rise to climate change vulnerability mapping. While not a new concept, the spatial analyses of specific health outcomes remain limited. This review explores different methodologies and data that are used to assess vulnerability and map population health impacts to climate hazards. The review retrieved scholarly articles and governmental reports concerning vulnerability mapping of human health to the impacts of climate change in the United States, published in the …


Influence Of Biological Sex On Social Behavior, Individual Recognition, And Non-Associative Learning In The Adult Gray Short-Tailed Opossum (Monodelphis Domestica), Mario Gil, Annelyn Torres-Reveron, Ana C. Ramirez, Oscar Maldonado, John L. Vandeberg, Gabriel A. De Erausquin Aug 2019

Influence Of Biological Sex On Social Behavior, Individual Recognition, And Non-Associative Learning In The Adult Gray Short-Tailed Opossum (Monodelphis Domestica), Mario Gil, Annelyn Torres-Reveron, Ana C. Ramirez, Oscar Maldonado, John L. Vandeberg, Gabriel A. De Erausquin

School of Medicine Publications

Social behavior is critical for relationship formation and is influenced by myriad environmental and individual factors. Basic and preclinical research typically relies on rodent models to identify the mechanisms that underlie behavior; however, it is important to use non-rodent models as well. A major objective of the present study was to test the hypothesis that biological sex and social experience modulate the expression of social behavior in the adult gray short-tailed opossum (Monodelphis domestica), a non-traditional model. We also investigated the non-associative learning abilities of these animals. Following a period of social isolation, animals of both sexes were …


Medical School Watercooler Newsletter - August 25, 2019, Lindsey Lyle Aug 2019

Medical School Watercooler Newsletter - August 25, 2019, Lindsey Lyle

Watercooler Newsletter

This is the August 25, 2019 edition of the Frederick P. Whiddon College of Medicine's Newsletter - Watercooler.

Contents Include:

USA medical students named to Alpha Omega Alpha Honor Medical Society

Macaluso named professor and chair of microbiology and immunology at USA College of Medicine

Clyde G. Huggins Award for Summer Research winners announced

Mark Your Calendar: Randall W. Powell Endowed Memorial Lectureship

Block party for a cause: increasing vaccination rates in rural Alabama

USA Health welcomes new interventional cardiologist


Cost Differences Between Sugammadex And Neostigmine Use In Non-Operating Room Anesthesia Care, Lindsey Marie Kilsdonk, Mindy Ruan Aug 2019

Cost Differences Between Sugammadex And Neostigmine Use In Non-Operating Room Anesthesia Care, Lindsey Marie Kilsdonk, Mindy Ruan

College of Science and Health Theses and Dissertations

rior to the introduction of sugammadex, the only option to reverse rocuronium-induced neuromuscular blockade (NMB) were cholinesterase inhibitors (i.e. neostigmine) and anticholinergics (i.e. glycopyrrolate). Sugammadex administration has been shown to provide a more effective and complete reversal of rocuronium. However, the higher cost of sugammadex has been cited as a limiting factor to its use. The medication costs of sugammadex and neostigmine with glycopyrrolate have been studied in the operating room setting. There is a lack of information regarding the usage and medication costs of sugammadex and neostigmine with glycopyrrolate for non-operating room anesthesia care (NORA). A retrospective chart review …


Would You Test For 5000 Shillings? Hiv Risk And Willingness To Accept Hiv Testing In Tanzania, Jan Ostermann, Derek S. Brown, Axel Mühlbacher, Bernard Njau, Nathan Thielman Aug 2019

Would You Test For 5000 Shillings? Hiv Risk And Willingness To Accept Hiv Testing In Tanzania, Jan Ostermann, Derek S. Brown, Axel Mühlbacher, Bernard Njau, Nathan Thielman

Faculty Publications

Objectives: Despite substantial public health efforts to increase HIV testing, testing rates have plateaued in many countries and rates of repeat testing for those with ongoing risk are low. To inform policies aimed at increasing uptake of HIV testing, we identified characteristics associated with individuals’ willingness-to-accept (WTA) an HIV test in a general population sample and among two high-risk populations in Moshi, Tanzania.

Methods: In total, 721 individuals, including randomly selected community members (N = 402), female barworkers (N = 135), and male Kilimanjaro mountain porters (N = 184), were asked in a double-bounded contingent valuation format if they would …


Certified Registered Nurse Anesthetist’S Awareness And Knowledge Of Herbal Supplements And Perioperative Interactions, Catherine Carman, Victoria N. Rosinski Aug 2019

Certified Registered Nurse Anesthetist’S Awareness And Knowledge Of Herbal Supplements And Perioperative Interactions, Catherine Carman, Victoria N. Rosinski

College of Science and Health Theses and Dissertations

Previous studies have asserted that anesthesia providers are deficient in their knowledge of herbal supplements and their perioperative interactions. The purpose of this project was to examine the impact of an educational handout on Certified Registered Nurse Anesthetists’ (CRNAs) knowledge and attitudes of herbal supplements and their perioperative interactions utilizing a pre-posttest project design. The survey components were sent to approximately 1,600 members of the Illinois Association of Nurse Anesthetists (IANA) with a response of 140 surveys. Data was analyzed using descriptive Cohen’s d and nonparametric statistical tests. Reliability of the instruments used were assessed using Cronbach’s ɑ coefficients. The …


Improving Knowledge And Self-Efficacy In Student Registered Nurse Anesthetists Through Simulation Based Learning On Pulmonary Artery Catheter Insertions, Neal Allen Johnson, Amanda Lipa Aug 2019

Improving Knowledge And Self-Efficacy In Student Registered Nurse Anesthetists Through Simulation Based Learning On Pulmonary Artery Catheter Insertions, Neal Allen Johnson, Amanda Lipa

College of Science and Health Theses and Dissertations

Simulation is increasingly being used within the educational setting for invasive procedures, such as pulmonary artery (PA) catheter insertion. The purpose of this study was to assess how student registered nurse anesthetists (SRNAs) knowledge and confidence is effected by viewing an educational video and participating in a low-fidelity simulation. Dual coding theory was used to develop a single group pre-test and post-test study. A convenience sample of second year SRNAs was used. Participants completed a knowledge assessment tool and a confidence survey. Each participant then viewed the video and completed a hands on low-fidelity simulation. Following both these, each participant …


Cultural Competence In Student Registered Nurse Anesthetists In Illinois, Lisa Brown, Saad Iqbal Aug 2019

Cultural Competence In Student Registered Nurse Anesthetists In Illinois, Lisa Brown, Saad Iqbal

College of Science and Health Theses and Dissertations

Objective: To determine the level of perceived cultural competence in student registered nurse anesthetists (SRNAs) in Illinois and make educational recommendations. Methods: A descriptive, cross-sectional study was completed using the Clinical Cultural Competency Questionnaire (CCCQ) to evaluate the perceived level of cultural competence among SRNAs in Illinois. Four domains of clinical cultural competency [knowledge, skills, attitudes (awareness), and encounters] were evaluated and reported. Results: The survey response rate was 16.7% (N=57). A statistically significant positive correlation was observed between cultural knowledge and age (p=0.03). There is a statistically significant difference in cultural knowledge between students attending a nurse anesthesia program …


Anesthesia Alarm Fatigue Policy Recommendations: The Path Of Development, Kelsey J. Leveille, Kelsey Anne Simoens Aug 2019

Anesthesia Alarm Fatigue Policy Recommendations: The Path Of Development, Kelsey J. Leveille, Kelsey Anne Simoens

College of Science and Health Theses and Dissertations

Healthcare workers are inundated with alarms every minute, yet 80-95% of these alarms do not result in provider intervention. False alarms cause a “cry-wolf” phenomenon among providers resulting in cognitive stress and workflow interruptions. A cross-sectional design was used to examine the perceptions of alarm fatigue and alarm management of Certified Registered Nurse Anesthetists (CRNAs), Student Registered Nurse Anesthetists (SRNAs) and Anesthesiologists. This Likert-scale questionnaire was sent to approximately 150 anesthesia providers at NorthShore University HealthSystem (NSUHS) through an online survey. Data analysis revealed anesthesia trainees and providers with less total years in practice and less clinical experience exhibit statistically …


Examination Of Reflections From Nurse Anesthesia Trainee Volunteers In Honduras, Thomas M. Boyce Aug 2019

Examination Of Reflections From Nurse Anesthesia Trainee Volunteers In Honduras, Thomas M. Boyce

College of Science and Health Theses and Dissertations

There is a lack of surgical and anesthesia services in low-income and middle-income countries (LMICs) which creates an increased burden of disease from otherwise surgically treatable conditions. Nurse anesthesia trainees (NATs) can serve to fill this lack of anesthesia services while also gaining real life experience that can enhance their training. Identifying common experiences and barriers among NATs that have volunteered on a surgical brigade is important to understand the benefits experienced by NATs and to increase volunteer surgical brigade programs among nurse anesthesia programs. A retrospective thematic analysis of 23 written reflections of NATs who participated in a one-week …


Identifying Pediatric Obesity In The Retail Health Setting And Implications For Quality, Jodie Nicole Guardi Aug 2019

Identifying Pediatric Obesity In The Retail Health Setting And Implications For Quality, Jodie Nicole Guardi

College of Science and Health Theses and Dissertations

Pediatric obesity has reached an epidemic status. The purpose of this study was to examine the prevalence of overweight and obese patients, as well as how often and how accurately a diagnosis was coded. The population for this study included children ages 3-18. This cohort presented to retail health clinics in a large urban health system for school or sports physicals from June 1, 2017 to November 30, 2017. Method. A retrospective chart review was conducted. Statistical analysis was performed to determine if there was a relationship between obesity and age, ethnicity, race, and gender. Results. The study identified 15.9% …


Pre-Anesthetic Evaluation: A Needs Assessment Of Student Registered Nurse Anesthetists Transitioning To Clinical Practice, Amanda B. Munnich, Cara A. Murphy Aug 2019

Pre-Anesthetic Evaluation: A Needs Assessment Of Student Registered Nurse Anesthetists Transitioning To Clinical Practice, Amanda B. Munnich, Cara A. Murphy

College of Science and Health Theses and Dissertations

The pre-anesthetic evaluation (PAE) is a critical part of providing anesthesia, and an important component of patient safety. Student registered nurse anesthetists (SRNAs) are often required to independently perform the PAE upon entering their clinical training. Stress, anxiety, doubt, a novel environment and time constraints can lead to a lack of confidence and competence at this time. These barriers can result in an inefficient and inadequate assessment. The purpose of this qualitative study was to conduct a needs assessment to determine the overall readiness of SRNAs to independently perform a thorough PAE upon entering clinical residency. A focus group was …


What We Say Matters: A Survey Of Anesthesia Providers’ Knowledge And Beliefs, Stefanie Glasgow, Lindsey Harris Aug 2019

What We Say Matters: A Survey Of Anesthesia Providers’ Knowledge And Beliefs, Stefanie Glasgow, Lindsey Harris

College of Science and Health Theses and Dissertations

Pain is a serious concern and fear of patients. Due to the Joint Commission’s introduction of standards to address undertreatment of pain, it is now a focus of healthcare providers. Emerging research is focused on language in assessing pain. This study investigated the knowledge gap among anesthesia providers on positive language and pain assessment. The study objectives were to conduct a survey of Illinois Association of Nurse Anesthetists (IANA) members’ current knowledge and beliefs on Positive Language and the Nocebo Effect related to patient outcomes, and to educate anesthesia providers with an evidence-based fact sheet. The fact sheet and Qualtrics …


Exploring The Protective Effects Of Judaism On Risky Behaviors In College Students, Michelle E. Neuman Aug 2019

Exploring The Protective Effects Of Judaism On Risky Behaviors In College Students, Michelle E. Neuman

College of Science and Health Theses and Dissertations

Background: Common risky behaviors among college-aged young adults include risky sexual behaviors and substance use. Purpose: This study examines the protective effects of Judaism on students’ engagement in risky behaviors, building on a body of research on the protective effects of religious beliefs on risky health behaviors. Methods: Validated and reliable measures were used to assess religiosity and risky behaviors through anonymous surveys. Data was collected in Fall 2018 from a small cohort (N=15) of Jewish day school students. Results: Higher religiosity was more associated with delayed sexual activity than substance use behaviors. Judaism and religiosity were more strongly associated …


Resident Physicians’ Perceptions Of Ambulatory Care Pharmacy, Ashley Meredith, Darin Ramsey, Andrew Schmelz, Rachel Berglund Aug 2019

Resident Physicians’ Perceptions Of Ambulatory Care Pharmacy, Ashley Meredith, Darin Ramsey, Andrew Schmelz, Rachel Berglund

Scholarship and Professional Work – COPHS

Background: Physicians' acceptance of clinical pharmacy services is dependent on exposure to those services, with use increasing as resident physicians progress through their training. Resident physicians train within environments that have a multidisciplinary teaching and clinical care approach, working closely with other healthcare professionals. Ambulatory care pharmacists are increasingly working with resident physicians in clinic settings as part of the multidisciplinary team, and identification of resident physicians’ perceptions may influence future collaboration.

Objective: The objective of this research is to evaluate the perception of ambulatory care clinical pharmacy services from the perspective of resident physicians.

Methods: A statewide network of …


A Renal Cell Carcinoma With Biallelic Somatic Tsc2 Mutation: Clinical Study And Literature Review., Jian Pang, Linang Wang, Jing Xu, Qiubo Xie, Qiuli Liu, Dali Tong, Gaolei Liu, Yiqiang Huang, Xingxia Yang, Jinhong Pan, Xiaochu Yan, Qiang Ma, Dianzheng Zhang, Jun Jiang Aug 2019

A Renal Cell Carcinoma With Biallelic Somatic Tsc2 Mutation: Clinical Study And Literature Review., Jian Pang, Linang Wang, Jing Xu, Qiubo Xie, Qiuli Liu, Dali Tong, Gaolei Liu, Yiqiang Huang, Xingxia Yang, Jinhong Pan, Xiaochu Yan, Qiang Ma, Dianzheng Zhang, Jun Jiang

PCOM Scholarly Works

OBJECTIVES: To elucidate the effect of the biallelic somatic TSC2 mutations, identified in one adolescent patient, in renal cell carcinoma (RCC).

METHODS: Mutation analyses, immunohistochemistry and real-time polymerase chain reaction (PCR) were conducted.

RESULTS: Two novel somatic mutations of TSC2 in unilateral and solitary RCC samples from a 14-year-old female were identified. The pathological features suggest the tumor as a clear-cell renal cell carcinoma. In addition, immunohistochemistry revealed elevated levels of phosphorylated S6K1. Results from in vitro cellular experiments suggest that the mutant TSC2 proteins were quickly degraded and they failed to repress the phosphorylation of S6K1 and STAT3, which …


Enhancing Patient Well-Being: Apply Positive Psychology In Nursing Practice, Farida Bibi Mughal Aug 2019

Enhancing Patient Well-Being: Apply Positive Psychology In Nursing Practice, Farida Bibi Mughal

School of Nursing & Midwifery

Objective: Positive psychology is the emerging concept in the medical field and has shown the positive effects on one’s well-being. This paper highlights the application of PERMA model in nursing practice.
Method: The model applied in the clinical area to access improvement in patient’s well-being. The concept of positive psychology’s applicability is discussed with the real life examples.
Result: The PERMA model when applied yields a positive impact on patient’s health and overall well-being.
Conclusion: The learning outcomes of this research will help the people in health care to understand the relationship between positive psychology and nursing practices for the …


Vascular Health And Mental Health In Collegiate Female Varsity Athletes, Club Sport Athletes And Recreationally Active Women, Allison Keller Aug 2019

Vascular Health And Mental Health In Collegiate Female Varsity Athletes, Club Sport Athletes And Recreationally Active Women, Allison Keller

Theses - ALL

The number of women participating in collegiate athletics has increased since Title IX was passed, which supports equal access to men and women in government-funded programs. Female student athletes are under increased pressure to maintain fitness while meeting academic standards, thus resulting in mental health concerns (i.e., depression and anxiety) and related poor lifestyle habits (i.e. problematic alcohol drinking and disturbed sleep quality). While high fitness imparts cardiovascular health benefits, such mental health concerns and poor lifestyle habits negatively impact cardiovascular health. Cardiovascular health is particularly concerning for women, as cardiovascular disease is a leading cause of death in this …


Identifying Opportunities For The Role Of Occupational Therapy In A Correctional Setting, Maxwell Sand Aug 2019

Identifying Opportunities For The Role Of Occupational Therapy In A Correctional Setting, Maxwell Sand

Student Capstone Papers

This study provides insight into the experiences of 7 individuals who had previous first-hand experience working in a correctional setting. Workplace culture related to health and wellness are discussed where participants disclose negative effects the environment posed to their well-being which in turn impacted their job satisfaction. Correctional settings prove not only to be restrictive to inmates, but also to staff members as they limit access to meaningful occupations, roles, and routines. Job demands linked to poor work policies and interaction with inmates are also found to take a toll physically, mentally, and emotionally which further exacerbates symptoms of stress, …


Resident Physicians’ Perceptions Of Ambulatory Care Pharmacy, Ashley H. Meredith, Darin Ramsey, Andrew Schmelz, Rachel Berglund Aug 2019

Resident Physicians’ Perceptions Of Ambulatory Care Pharmacy, Ashley H. Meredith, Darin Ramsey, Andrew Schmelz, Rachel Berglund

Department of Pharmacy Practice Faculty Publications

Background: Physicians' acceptance of clinical pharmacy services is dependent on exposure to those services, with use increasing as resident physicians progress through their training. Resident physicians train within environments that have a multidisciplinary teaching and clinical care approach, working closely with other healthcare professionals. Ambulatory care pharmacists are increasingly working with resident physicians in clinic settings as part of the multidisciplinary team, and identification of resident physicians’ perceptions may influence future collaboration.

Objective: The objective of this research is to evaluate the perception of ambulatory care clinical pharmacy services from the perspective of resident physicians.

Methods: A statewide network of …


Exploring The Role Of Occupational Therapy In Primary Care, Tina Rosselli Aug 2019

Exploring The Role Of Occupational Therapy In Primary Care, Tina Rosselli

Student Capstone Papers

Background: With an increase in adult chronic conditions (Holstein, 2018), as well as a growing number of older adults aging in place (Dickson & Toto, 2018), primary care will continue to see the greatest number of patients as it is the first point of contact for a person to enter the health-care system. Most primary care clinics are reimbursed based on quality outcome measures, which means insurance payers are looking at the value of care instead of the quantity of services (Halle et al., 2018). There is value in as well as an absence of assessment of occupational participation …


Screening And Diagnosing Spinal Muscular Atrophy By Use Of Buccal Swabs: A Validation Study Using Ddpcr, Anna Mackay Aug 2019

Screening And Diagnosing Spinal Muscular Atrophy By Use Of Buccal Swabs: A Validation Study Using Ddpcr, Anna Mackay

Honors Scholar Theses

Spinal Muscular Atrophy (SMA) is a genetic neuromuscular disorder characterized by progressive muscle weakness due to the degeneration of motor neurons. SMA is caused by a homozygous deletion, mutation or rearrangement in the Survival Motor Neuron 1 (SMN1) gene. Survival Motor Neuron 2 (SMN2) is located tandem to SMN1 and is identical to SMN1 except for a single nucleotide substitution in exon 7. SMA diagnosis and carrier status can be determined by droplet digital PCR (ddPCR). This study sought to validate Bio-Rad’s ddPCR SMN1and SMN2 gene determination copy number assay for SMA diagnosis and screening using buccal swabs specimens. Buccal …


Hope, Courage, And Resilience In The Lives Of Transgender Women Of Color, Nadine Ruff, Amy B. Smoyer, Jean Breny Aug 2019

Hope, Courage, And Resilience In The Lives Of Transgender Women Of Color, Nadine Ruff, Amy B. Smoyer, Jean Breny

The Qualitative Report

There is a lack of qualitative and strengths-based knowledge about the lived experience of transgender women of color in the US. To address this research gap, a Photovoice project was undertaken with five transgender women living in a small urban area. Thematic analysis of the participants’ discussion of their photographs identified three major themes: hope, courage, and resilience. Analysis suggests a framework for understanding these women’s lived experiences and the psychosocial tools that they use to negotiate their daily lives and persevere in the face of interpersonal and structural oppression.


Development Of Substrate Degradation Enzyme Therapy For Mucopolysaccharidosis Iva Murine Model., Kazuki Sawamoto, Shunji Tomatsu Aug 2019

Development Of Substrate Degradation Enzyme Therapy For Mucopolysaccharidosis Iva Murine Model., Kazuki Sawamoto, Shunji Tomatsu

Department of Pediatrics Faculty Papers

Mucopolysaccharidosis IVA (MPS IVA) is caused by a deficiency of the lysosomal enzyme N-acetylgalactosamine-6-sulfate sulfatase (GALNS). Conventional enzyme replacement therapy (ERT) is approved for MPS IVA. However, the fact that the infused enzyme cannot penetrate avascular lesions in cartilage leads to minimal impact on the bone lesion. Moreover, short half-life, high cost, instability, and narrow optimal pH range remain unmet challenges in ERT. Thermostable keratanase, endo-β-N-acetylglucosaminidase, has a unique character of a wide optimal pH range of pH 5.0-7.0. We hypothesized that this endoglycosidase degrades keratan sulfate (KS) polymer in circulating blood and, therefore, ameliorates the accumulation of KS in …