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A Rationally Engineered Capsid Variant Of Aav9 For Systemic Cns-Directed And Peripheral Tissue-Detargeted Gene Delivery In Neonates, Dan Wang, Shaoyong Li, Dominic J. Gessler, Jun Xie, Li Zhong, Jia Li, Karen Tran, Kim Van Vliet, Lingzhi Ren, Qin Su, Ran He, Jason E. Goetzmann, Terence R. Flotte, Mavis Agbandje-McKenna, Guangping Gao 2018 University of Massachusetts Medical School

A Rationally Engineered Capsid Variant Of Aav9 For Systemic Cns-Directed And Peripheral Tissue-Detargeted Gene Delivery In Neonates, Dan Wang, Shaoyong Li, Dominic J. Gessler, Jun Xie, Li Zhong, Jia Li, Karen Tran, Kim Van Vliet, Lingzhi Ren, Qin Su, Ran He, Jason E. Goetzmann, Terence R. Flotte, Mavis Agbandje-Mckenna, Guangping Gao

Open Access Articles

Adeno-associated virus (AAV) has provided the gene therapy field with the most powerful in vivo gene delivery vector to realize safe, efficacious, and sustainable therapeutic gene expression. Because many clinically relevant properties of AAV-based vectors are governed by the capsid, much research effort has been devoted to the development of AAV capsids for desired features. Here, we combine AAV capsid discovery from nature and rational engineering to report an AAV9 capsid variant, designated as AAV9.HR, which retains AAV9's capability to traverse the blood-brain barrier and transduce neurons. This variant shows reduced transduction in peripheral tissues when delivered through ...


Rates Of Developmental And Behavioral Screening Of Young Children: Implications For Health Care Policy And Practice, Shirley Berger 2018 CUNY School of Public Health

Rates Of Developmental And Behavioral Screening Of Young Children: Implications For Health Care Policy And Practice, Shirley Berger

Dissertations and Theses

Background: The skills and capacities developed during early childhood are the foundation for a child’s future academic functioning, economic productivity, and lifelong health and mental health. When young children have developmental delays or disabilities, early identification and intervention lead to better outcomes; however, only a minority are identified before school entry. Primary care is an important setting for identification of developmental and behavioral conditions as most young children attend well-child visits regularly and parents expect developmental guidance from pediatricians. Two key pediatric preventive services are recommended: developmental monitoring/surveillance at every well-child visit and developmental screening at 9 months ...


Effect Of Life Skills Building Education And Micronutrient Supplements Provided From Preconception Versus The Standard Of Care On Low Birth Weight Births Among Adolescent And Young Pakistani Women (15–24 Years): A Prospective, Population-Based Cluster-Randomized Trial, Jo-Anna B. Baxter, Yaqub Wasan, Sajid Bashir Soofi, Zamir Suhag, Zulfiqar Ahmed Bhutta 2018 University of Toronto

Effect Of Life Skills Building Education And Micronutrient Supplements Provided From Preconception Versus The Standard Of Care On Low Birth Weight Births Among Adolescent And Young Pakistani Women (15–24 Years): A Prospective, Population-Based Cluster-Randomized Trial, Jo-Anna B. Baxter, Yaqub Wasan, Sajid Bashir Soofi, Zamir Suhag, Zulfiqar Ahmed Bhutta

Department of Paediatrics and Child Health

Background: Risk factors known to impact maternal and newborn nutrition and health can exist from adolescence. If an undernourished adolescent girl becomes pregnant, her own health and pregnancy are at an increased risk for adverse outcomes. Offering preconception carefrom adolescence could provide an opportunity for health and nutrition promotion to improve one's own well-being, as well as future pregnancy outcomes and the health of the next generation.
Methods: The Matiari emPowerment and Preconception Supplementation (MaPPS) Trial is a population-based two-arm, cluster-randomized, controlled trial of life skills building education and multiple micronutrient supplementation provided in a programmatic context to evaluate ...


Feasibility And Effect Of Life Skills Building Education And Multiple Micronutrient Supplements Versus The Standard Of Care On Anemia Among Non-Pregnant Adolescent And Young Pakistani Women (15-24 Years): A Prospective, Population-Based Cluster-Randomized Trial, Jo-Anna B. Baxter, Yaqub Wasan, Sajid Bashir Soofi, Zamir Suhag, Zulfiqar Ahmed Bhutta 2018 The Hospital for Sick Children

Feasibility And Effect Of Life Skills Building Education And Multiple Micronutrient Supplements Versus The Standard Of Care On Anemia Among Non-Pregnant Adolescent And Young Pakistani Women (15-24 Years): A Prospective, Population-Based Cluster-Randomized Trial, Jo-Anna B. Baxter, Yaqub Wasan, Sajid Bashir Soofi, Zamir Suhag, Zulfiqar Ahmed Bhutta

Department of Paediatrics and Child Health

Background: Adolescence is a critical period for physical and psychological growth and development, and vitamin and mineral requirements are correspondingly increased. Health and health behaviours correspond strongly from adolescence to adulthood. Developing a preconception care package for adolescent and young women in resource-limited settings could serve to empower them to make informed decisions about their nutrition, health, and well-being, as well as function as a platform for the delivery of basic nutrition-related interventions to address undernutrition.
Methods: In this population-based two-arm, cluster-randomized, controlled trial of life skills building education (provided bi-monthly) and multiple micronutrient supplementation (provided twice-weekly; UNIMMAP composition), we ...


Reducing Pediatric Asthma Visits In The Emergency Department, Kathleen Grimley Ahn 2018 University of San Diego

Reducing Pediatric Asthma Visits In The Emergency Department, Kathleen Grimley Ahn

Doctor of Nursing Practice Final Manuscripts

Purpose/Aims: The purpose of this project was to reduce the number of unnecessary pediatric asthma management visits to an urban emergency department (ED) by performing a brief action plan for improved National Heart, Lung, and Blood Institute (NHLBI) guideline adherence. This plan was shared with parents during an NP-initiated follow-up phone call.

Rationale/Background: Inadequate outpatient management of pediatric asthma can result in avoidable visits to the ED. Acute exacerbations comprise a large portion of pediatric asthma patients seeking care in the ED. Reasons for persistent under-utilization of outpatient care for asthma management in primary care settings are multifactorial ...


Admission Eeg Findings In Diverse Paediatric Cerebral Malaria Populations Predict Outcomes., Douglas G Postels, Xiaoting Wu, Chenxi Li, Peter W Kaplan, Karl B Seydel, Terrie E Taylor, Youssef A Kousa, Richard Idro, Robert Opoka, Chandy C John, Gretchen L Birbeck 2018 George Washington University

Admission Eeg Findings In Diverse Paediatric Cerebral Malaria Populations Predict Outcomes., Douglas G Postels, Xiaoting Wu, Chenxi Li, Peter W Kaplan, Karl B Seydel, Terrie E Taylor, Youssef A Kousa, Richard Idro, Robert Opoka, Chandy C John, Gretchen L Birbeck

Pediatrics Faculty Publications

BACKGROUND: Electroencephalography at hospital presentation may offer important insights regarding prognosis that can inform understanding of cerebral malaria (CM) pathophysiology and potentially guide patient selection and risk stratification for future clinical trials. Electroencephalogram (EEG) findings in children with CM in Uganda and Malawi were compared and associations between admission EEG findings and outcome across this diverse population were assessed. Demographic, clinical and admission EEG data from Ugandan and Malawian children admitted from 2009 to 2012 with CM were gathered, and survivors assessed for neurological abnormalities at discharge.

RESULTS: 281 children were enrolled (Uganda n = 122, Malawi n = 159). The Malawian ...


Improved Growth And Development In Premature Infants Managed With Nasal Continuous Positive Airway Pressure, Susan L. Flesher M.D., Renee S. Domanico M.D. 2018 Marshall University

Improved Growth And Development In Premature Infants Managed With Nasal Continuous Positive Airway Pressure, Susan L. Flesher M.D., Renee S. Domanico M.D.

Susan L Flesher, MD

Objective: Our goal was to assess the association between the use of nasal continuous positive airway pressure (NCPAP) vs. conventional ventilation (CV) in premature infants and its effects on: 1) growth in the NICU and at follow up visits 2) neurodevelopmental outcomes measured by Bayley Infant Neurodevelopmental Screener (BINS) 3) the incidence of retinopathy of prematurity (ROP) and chronic lung disease (CLD).

Methods: A retrospective chart review of two groups of NICU patients was conducted. The first group was from 1/1999 – 12/2000 (n=140) and was managed by CV. The second group (n=168) was from 1/2003 ...


Facilitating The Everyday Steward: Impact Of Mandatory Antimicrobial Indication/Duration And A 48 Hour Time Out, Ann L. Wirtz, Alaina N. Burns, Brian R. Lee, Tammy Frank, Laura Fitzmaurice, Richard Ogden, Brian O'Neal, Jennifer Goldman 2018 Children's Mercy Kansas City

Facilitating The Everyday Steward: Impact Of Mandatory Antimicrobial Indication/Duration And A 48 Hour Time Out, Ann L. Wirtz, Alaina N. Burns, Brian R. Lee, Tammy Frank, Laura Fitzmaurice, Richard Ogden, Brian O'Neal, Jennifer Goldman

Posters

Introduction: Required indication, duration, and a 48-hour antimicrobial timeout are an integral part of antimicrobial stewardship standards; however, limited data are available to demonstrate an effect on antimicrobial utilization and antimicrobial stewardship practice. Therefore, we evaluated the impact of mandatory declared indication/duration along with a pharmacy-driven 48-hour timeout on antimicrobial utilization and antimicrobial stewardship interventions.

Methods: We performed a retrospective evaluation of ASP interventions and antimicrobial use following implementation of mandatory antimicrobial indication/duration at the point of computerized physician order entry (CPOE). A pharmacist-driven 48-hour antimicrobial timeout was introduced on the same date. This study was conducted at ...


Extraction And Analysis Of Vector Flow Imaging Data In A Pediatric Population, Bailey Stinnett 2018 University of Arkansas, Fayetteville

Extraction And Analysis Of Vector Flow Imaging Data In A Pediatric Population, Bailey Stinnett

Biomedical Engineering Undergraduate Honors Theses

Vector flow imaging (VFI) is a new ultrasound technology that provides real time, angle-independent visualization of flow velocities in the heart and great vessels. Thus far, VFI has been used for superficial applications due to the limited penetration depth of available transducer probes; however, this depth in smaller pediatric patients enables adequate aortic views. In this project, VFI was used to study pediatric aortic stenosis (PAS)—a congenital heart defect that results in the narrowing of the aorta and/or aortic valve. The decision to refer PAS patients for surgical or catheter-based intervention is initially based on Doppler ultrasound. VFI ...


Optimization Of The Resident Didactic Curriculum - High Yield, High Reward!, Abigail Klitsch DO, Kris Rooney MD 2018 Lehigh Valley Health Network

Optimization Of The Resident Didactic Curriculum - High Yield, High Reward!, Abigail Klitsch Do, Kris Rooney Md

Department of Pediatrics

No abstract provided.


Survey Of Sensory Diet Use Among California Occupational Therapy Practitioners, Morganne Peterson, Allison Hunt, Emily White 2018 Dominican University of California

Survey Of Sensory Diet Use Among California Occupational Therapy Practitioners, Morganne Peterson, Allison Hunt, Emily White

Master's Theses and Capstone Projects

The purpose of this study was to examine the use of sensory diets in the field of occupational therapy. This study investigated the use of sensory diets among California occupational therapy practitioners. A mixed-methods design was used to collect data through an online survey. The survey was sent out to members of the Occupational Therapy Association of California (OTAC) and received 98 respondents within one month. Participants worked among various clinical settings and implemented sensory diets with various client populations. Practitioners reported using terms such as “sensory strategies,” “sensory tools,” and “sensory supports” which indicates an overall misunderstanding associated with ...


Effect Of Trinucleotide Repeats In The Huntington's Gene On Intelligence, Jessica K. Lee, Amy Conrad, Eric Epping, Kathy Mathews, Vincent Magnotta, Jeffrey D. Dawson, Peg Nopoulos 2018 University of Iowa

Effect Of Trinucleotide Repeats In The Huntington's Gene On Intelligence, Jessica K. Lee, Amy Conrad, Eric Epping, Kathy Mathews, Vincent Magnotta, Jeffrey D. Dawson, Peg Nopoulos

Stead Family Department of Pediatrics Publications

BACKGROUND: Huntington's Disease (HD) is caused by an abnormality in the HTT gene. This gene includes trinucleotide repeats ranging from 10 to 35, and when expanded beyond 39, causes HD. We previously reported that CAG repeats in the normal range had a direct and beneficial effect on brain development with higher repeats being associated with higher cognitive function. The current study now expands this line of inquiry to evaluate the effects of CAG repeat throughout the entire spectrum of repeats from 15 to 58.

METHODS: We evaluated brain function in children ages 6-18 years old. DNA samples were processed ...


The Utility Of Routine Echocardiography In Newborn Infants With A Persistent Oxygen Requirement ., Niamh Walsh, C Breathnach, Afif F. El-Khuffash, Orla Franklin, John D. Corcoran 2018 Rotunda Hospital, Dublin

The Utility Of Routine Echocardiography In Newborn Infants With A Persistent Oxygen Requirement ., Niamh Walsh, C Breathnach, Afif F. El-Khuffash, Orla Franklin, John D. Corcoran

Department of Paediatrics Articles

In the era of antenatal screening for congenital heart disease (CHD), infants presenting with an undiagnosed significant CHD are rare. However, term infants admitted with an initial diagnosis of TTN and a prolonged oxygen requirement often undergo an echocardiogram. We aimed to assess whether this practice yields any additional cases of undiagnosed CHD. We performed a retrospective chart review over a three year period [2013 – 2015] of term (> 36 weeks) infants admitted to the NICU for ≥ 5 days with a diagnosis of TTN and received an echocardiogram. The presence of CHD on the echocardiogram was assessed. Forty-seven infants were enrolled ...


Childhood-Onset Systemic Lupus Erythematosus: A Cohort Study, Shakeel Ahmed, Syed Rehan Ali, Farida Karim, Sidra Ishaque, Iman Qaiser, Kanwal Nayani, Ayesha Ahmed 2018 Aga Khan University

Childhood-Onset Systemic Lupus Erythematosus: A Cohort Study, Shakeel Ahmed, Syed Rehan Ali, Farida Karim, Sidra Ishaque, Iman Qaiser, Kanwal Nayani, Ayesha Ahmed

Department of Paediatrics and Child Health

Objective: To determine the clinical and immunological characteristics and short-term outcome of children with systemic lupus erythematosus (SLE).
Study Design: A descriptive cohort study.
Place and Duration of Study: Paediatric Rheumatology Clinic, The Aga Khan University Hospital, Karachi, from January 2011 to December 2015.
Methodology: Clinical and immunological profile and short-term outcome of children less than 16 years of age admitted in the paediatric ward, with the diagnosis of SLE was studied. Demographic data, clinical presentation, laboratory findings, immunological profile and treatment regimens of these children were evaluated.
Results: Thirty-two children, satisfying the criteria of American College of Rheumatology (ACR ...


Amygdala Corticofugal Input Shapes Mitral Cell Responses In The Accessory Olfactory Bulb, Livio Oboti, Eleonora Russo, Tuyen Tran, Daniel Durstewitz, Joshua G. Corbin 2018 George Washington University

Amygdala Corticofugal Input Shapes Mitral Cell Responses In The Accessory Olfactory Bulb, Livio Oboti, Eleonora Russo, Tuyen Tran, Daniel Durstewitz, Joshua G. Corbin

Pediatrics Faculty Publications

Interconnections between the olfactory bulb and the amygdala are a major pathway for triggering strong behavioral responses to a variety of odorants. However, while this broad mapping has been established, the patterns of amygdala feedback connectivity and the influence on olfactory circuitry remain unknown. Here, using a combination of neuronal tracing approaches, we dissect the connectivity of a cortical amygdala [posteromedial cortical nucleus (PmCo)] feedback circuit innervating the mouse accessory olfactory bulb. Optogenetic activation of PmCo feedback mainly results in feedforward mitral cell (MC) inhibition through direct excitation of GABAergic granule cells. In addition, LED-driven activity of corticofugal afferents increases ...


Surfactant Deficiency Syndrome In An Infant With A C-Terminal Frame Shift In Abca3: A Case Report, Nour Akil, Anthony J. Fischer 2018 University of Iowa

Surfactant Deficiency Syndrome In An Infant With A C-Terminal Frame Shift In Abca3: A Case Report, Nour Akil, Anthony J. Fischer

Stead Family Department of Pediatrics Publications

Deficiency in ATP binding cassette A3 (ABCA3) causes neonatal respiratory distress, hypoxemic respiratory failure, and interstitial lung disease. ABCA3 transports phospholipids into the lamellar bodies of type II alveolar cells, a critical step in alveolar surfactant production. We report a term infant with ABCA3 surfactant deficiency syndrome with the E292V (c.875A>T; p.Glu292Val) mutation in trans with a novel C-terminal frame shift mutation (c.4938delC; p.Met1647fs). This mutation removes the final 58 amino acids and substitutes 33 incorrect amino acids. The frame shift spares membrane spanning and nucleotide binding domains, but disrupts a highly conserved C-terminal domain ...


Dystrophic Calcifications Point The Way-Unusual And Early Diagnostic Clue Of Conradi-Hünermann-Happle Syndrome, Kenneth Yu, Alexander T. Reid, Stephanie J T Chen, Rajiv M. Patel, Steven M. Donn, Johann E. Gudjonsson, Lori Lowe 2018 University of Iowa

Dystrophic Calcifications Point The Way-Unusual And Early Diagnostic Clue Of Conradi-Hünermann-Happle Syndrome, Kenneth Yu, Alexander T. Reid, Stephanie J T Chen, Rajiv M. Patel, Steven M. Donn, Johann E. Gudjonsson, Lori Lowe

Stead Family Department of Pediatrics Publications

No abstract provided.


Oxytocin Promotes Face-Sensitive Neural Responses To Infant And Adult Faces In Mothers, Mikko J. Peltola, Lane Strathearn, Kaija Puura 2018 University of Iowa

Oxytocin Promotes Face-Sensitive Neural Responses To Infant And Adult Faces In Mothers, Mikko J. Peltola, Lane Strathearn, Kaija Puura

Stead Family Department of Pediatrics Publications

Research utilizing intranasal oxytocin (OT) administration has shown that OT may increase attention and sensitivity to social cues, such as faces. Given the pivotal role of OT in parental behaviors across mammals, the paucity of intranasal OT research investigating responses to social cues in parents and particularly mothers of young children is a critical limitation. In the current study, we recorded cortical event-related potentials (ERPs) to investigate whether intranasal OT affects the early neural responses to emotional faces in mothers of 1-year-old infants. Using a double-blind, within-subjects design, mothers (n = 38) were administered intranasal OT and placebo on separate sessions ...


Prevalence Of Prediabetes, Diabetes, And Its Associated Risk Factors Among Males In Saudi Arabia: A Population-Based Survey, Khaled K. Aldossari, Abdulrahman Aldiab, Jamaan M. Al-Zahrani, Sameer H. Al-Ghamdi, Mohammed Abdelrazik, Mohammed Ali Batais, Sundas Javad, Shanila Nooruddin, Hira Abdul Razzak, Ashraf El-Metwally 2018 Prince Sattam Bin Abdulaziz University

Prevalence Of Prediabetes, Diabetes, And Its Associated Risk Factors Among Males In Saudi Arabia: A Population-Based Survey, Khaled K. Aldossari, Abdulrahman Aldiab, Jamaan M. Al-Zahrani, Sameer H. Al-Ghamdi, Mohammed Abdelrazik, Mohammed Ali Batais, Sundas Javad, Shanila Nooruddin, Hira Abdul Razzak, Ashraf El-Metwally

Department of Paediatrics and Child Health

Objectives: The study aims at determining the prevalence of prediabetes and diabetes and at ascertaining some concomitant risk factorsamong males in Saudi Arabia.
Methods: A population-based cross-sectional study including 381 Saudi adult males from different institutions was recruited. Odds ratios for diabetes risk and risk factors were calculated using log-binomial and multinomial logistic regression, using STATA version 12.
Results: The participants included 381 diabetic males with a median age of 45 years, average body mass index of 25 ± 40 kg/m2, whereas waist circumferences ranged from 66 to 180 cm in the male study population. In addition, 27.82 ...


A Global Health Research Checklist For Clinicians., Rasha D Sawaya, Kristen A Breslin, Eiman Abdulrahman, Jennifer I Chapman, Dafina M Good, Lili Moran, Paul C Mullan, Oluwakemi Badaki-Makun 2018 George Washington University

A Global Health Research Checklist For Clinicians., Rasha D Sawaya, Kristen A Breslin, Eiman Abdulrahman, Jennifer I Chapman, Dafina M Good, Lili Moran, Paul C Mullan, Oluwakemi Badaki-Makun

Pediatrics Faculty Publications

Global health research has become a priority in most international medical projects. However, it is a difficult endeavor, especially for a busy clinician. Navigating the ethics, methods, and local partnerships is essential yet daunting.To date, there are no guidelines published to help clinicians initiate and complete successful global health research projects. This Global Health Research Checklist was developed to be used by clinicians or other health professionals for developing, implementing, and completing a successful research project in an international and often low-resource setting. It consists of five sections: Objective, Methodology, Institutional Review Board and Ethics, Culture and partnerships, and ...


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