The faculty members of the Department of Pediatrics at the University of Massachusetts Medical School teach medical students, interns and residents, and they conduct research in many topics related to child health such as HIV, leukemia, diabetes, cystic fibrosis, teen pregnancy, infant mortality, and health care delivery in the community. The University of Massachusetts Medical School is one of about 40 centers for the National Children’s Study (NCS), a federally funded longitudinal study to investigate the effects of the environment on the growth and development and health of children by following 100,000 children from pre-birth to age 21 years. Approximately 1000 of those children will be identified from Worcester County residents in Massachusetts. UMass is the only institution in Massachusetts to be identified as an NCS Center. This collection showcases the journal articles and other publications authored by faculty and researchers in the Department of Pediatrics.

To view this collection broken out by Department of Pediatrics divisions, visit Pediatric Divisions.

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Publications from 2021

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Association of Maternal Perinatal SARS-CoV-2 Infection With Neonatal Outcomes During the COVID-19 Pandemic in Massachusetts, Asimenia Angelidou, Katherine P. Sullivan, Patrice R. Melvin, Jessica E. Shui, Ilona Telefus Goldfarb, Ruby Bartolome, Neha Chaudhary, Ruben Vaidya, Ivana Culic, Rachana Singh, Diana Yanni, Silvia Patrizi, Mark L. Hudak, Margaret G. Parker, and Mandy B. Belfort

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Lack of Progression of Intraventricular Hemorrhage in Premature Infants: Implications for Head Ultrasound Screening, Jaclyn Daigneault, Heather White, Alexandra Dube, Qiming Shi, Jean-Marc Gauguet, and Lawrence M. Rhein

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Automatic referrals within a cystic fibrosis multidisciplinary clinic improve patient evaluation and management, Amy Darukhanavala and Ted M. Kremer

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Use of hemoglobin A1c to identify dysglycemia in cystic fibrosis, Amy Darukhanavala, Filia Van Dessel, Jannifer Ho, Megan Hansen, Ted M. Kremer, and David Alfego

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Recent Advances in miRNA Delivery Systems, Ishani Dasgupta and Anushila Chatterjee

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A Series of Patients With Myocarditis Following SARS-CoV-2 Vaccination With mRNA-1279 and BNT162b2, John B. Dickey, Elisabeth Albert, Mai Badr, Kristin M. Laraja, Laureen M. Sena, David S. Gerson, Jason E. Saucedo, Waqas Qureshi, and Gerard P. Aurigemma

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Viral Loads of SARS-CoV-2 in Young Children, Heather Finlay-Morreale

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Liver targeting with rAAV7: balancing tropism with immune profiles, Terence R. Flotte

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In Reply to Ramotshwana et al, Terence R. Flotte, Anne C. Larkin, Melissa A. Fischer, Sonia N. Chimienti, Deborah M. DeMarco, Pang-Yen Fan, and Michael F. Collins

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The RADx Tech Clinical Studies Core: A Model for Academic Based Clinical Studies, Laura L. Gibson, Nisha M. Fahey, Nathaniel Hafer, John P. Broach, Bruce A. Barton, Stephenie C. Lemon, Allison Blodgett, and David D. McManus

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Should treatment of low-level rifampicin mono-resistant tuberculosis be different, F. A. Gopie, E. Commiesie, S. Baldi, M. Kamst, Devinder Kaur, W. C. M. de Lange, P. S. Pinas, D. Stijnberg, M. Wongsokarijo, C. W. R. Zijlmans, R. de Zwaan, D. van Soolingen, S. G. S. Vreden, and G. de Vries

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Massachusetts' Findings from Statewide Newborn Screening for Spinal Muscular Atrophy, Jaime E. Hale, Basil T. Darras, Kathryn J. Swoboda, Elicia Estrella, Jin Yun Helen Chen, Mary-Alice Abbott, Beverly N. Hay, Binod Kumar, Anne M. Counihan, Jacalyn L. Gerstel-Thompson, Inderneel Sahai, Roger B. Eaton, and Anne Marie Comeau

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Common pathways to Dean of Medicine at U.S. medical schools, Clare E. Jacobson, Whitney H. Beeler, Kent A. Griffith, Terence R. Flotte, Carrie L. Byington, and Reshma Jagsi

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Cannabis Product Ingestions in Pediatric Patients: Ranges of Exposure, Effects, and Outcomes, Eric E. Kaczor, Bonnie L. Mathews, Kara L. LaBarge, Brittany Chapman, and Stephanie Carreiro

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Child Care During the COVID-19 Pandemic: A Bad Situation Made Worse, Nikita Kalluri, Colleen Kelly, and Arvin Garg

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Physician Perspectives about Telemedicine: Considering the Usability of Telemedicine in Response to COVID-19, Jennifer A. Lee, Gennaro Di Tosto, Fiona A. McAlearney, Steven Miller, Ethan Mezoff, Rajitha D. Venkatesh, Jeannie Huang, Jenifer R. Lightdale, Jaclyn Volney, and Ann Scheck. McAlearney

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Novel Combinatorial MicroRNA-Binding Sites in AAV Vectors Synergistically Diminish Antigen Presentation and Transgene Immunity for Efficient and Stable Transduction, Manish Muhuri, Wei Zhan, Yukiko Maeda Garrison, Jia Li, Anoushka Lotun, Jennifer Chen, Katelyn E. Sylvia, Ishani Dasgupta, Motahareh Arjomandnejad, Thomas Nixon, Allison M. Keeler, Sangeetha Manokaran, Ran He, Qin Su, Phillip W. L. Tai, and Guangping Gao

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Supplementary materials for: Ergocalciferol in New-onset Type 1 diabetes: A Randomized Controlled Trial, Benjamin U. Nwosu

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COVID-19 Pandemic and Pediatric Type 1 Diabetes: No Significant Change in Glycemic Control During The Pandemic Lockdown of 2020, Benjamin U. Nwosu, Layana Al-Halbouni, Sadichchha Parajuli, Gabrielle Jasmin, Emily Zitek-Morrison, and Bruce A. Barton

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Long-term GH Therapy Does Not Advance Skeletal Maturation in Children and Adolescents, Benjamin U. Nwosu, Gabrielle Jasmin, Sadichchha Parajuli, Alan D. Rogol, Ellen C. Wallace, and Austin F. Lee

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Ergocalciferol in New-onset Type 1 diabetes: A Randomized Controlled Trial, Benjamin U. Nwosu, Sadichchha Parajuli, Gabrielle Jasmin, Jody Fleshman, Rohit B. Sharma, Laura C. Alonso, Austin F. Lee, and Bruce A. Barton

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Supporting families of children with overweight and obesity to live healthy lifestyles: Design and rationale for the Fitline cluster randomized controlled pediatric practice-based trial, Lori Pbert, Michelle K. Trivedi, Susan Druker, Jennifer Bram, Barbara C. Olendzki, Sybil L. Crawford, Christine F. Frisard, Victoria A. Andersen, Molly E. Waring, Karen M. Clements, Kristin Schneider, and Alan C. Geller

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Racial and Ethnic Differences in Managed Care Enrollment Among US Children, Alon Peltz, Kristin Kan, Arvin Garg, Alexander Pomerantz, Lucy A. Bilaver, and Matthew M. Davis

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Improved prime editors enable pathogenic allele correction and cancer modelling in adult mice, Pengpeng Liu, Shun-Qing Liang, Chunwei Zheng, Esther Mintzer, Yan G. Zhao, Karthikeyan Ponnienselvan, Aamir Mir, Erik J. Sontheimer, Guangping Gao, Terence R. Flotte, Scot A. Wolfe, and Wen Xue

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Up-regulation of miR-34b/c by JNK and FOXO3 protects from liver fibrosis, Pasquale Piccolo, Florie Borel, Christian Mueller, and Nicola Brunetti-Pierri

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Study design and rationale for a randomized controlled trial to assess effectiveness of stochastic vibrotactile mattress stimulation versus standard non-oscillating crib mattress for treating hospitalized opioid-exposed newborns, Elisabeth B. Salisbury, Debra Bogen, Mark A. Vining, Dane Netherton, Nicolas Rodriguez, Tory Bruch, Cheryl Burns, Emily Erceg, Barbara Glidden, M. Didem Ayturk, Sanjay Aurora, Toby Yanowitz, Bruce A. Barton, and Sue Beers

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Longitudinal assessment of diagnostic test performance over the course of acute SARS-CoV-2 infection, Rebecca L. Smith, Laura L. Gibson, Alyssa N. Owens, John P. Broach, Bruce A. Barton, Peter Lazar, David D. McManus, and Christopher B. Brooke

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Adapting Helping Babies Breathe into a Virtual Curriculum: Methods, Results, and Lessons Learned, Celia Sobelman, Kristen Richard, Patricia A. McQuilkin, and Nisha Fahey

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Transcutaneous carbon dioxide pattern and trend over time in preterm infants, Katherine P. Sullivan, Heather White, Lindsay E. Grover, Jordi J. Negron, Austin F. Lee, and Lawrence M. Rhein

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Leveraging the Massachusetts perinatal quality collaborative to address the COVID-19 pandemic among diverse populations, Katherine Sullivan, Ruben Vaidya, and Rachana Singh

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Incorporating systems-level stakeholder perspectives into the clinical trial design of school-supervised asthma therapy, Michelle Trivedi, Shushmita Hoque, Janki Luther, Michelle Spano, Holly Shillan, Hallie Pearl, Hannah L. Seay, Wanda Phipatanakul, Lynn B. Gerald, and Lori Pbert

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Is this the end for ranitidine? NDMA presence continues to confound, Jennifer A. Wagner, Jean C. Dinh, Jenifer R. Lightdale, Benjamin D. Gold, and Jennifer M. Colombo

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The Intestinal and Oral Microbiomes Are Robust Predictors of COVID-19 Severity the Main Predictor of COVID-19-related Fatality [preprint], Doyle V. Ward, Shakti Bhattarai, Mayra Rojas-Correa, Ayan Purkayastha, Devon Holler, Ming Da Qu, William G. Mitchell, Jason D. Yang, Samuel Fountain, Abigail Zeamer, Catherine Forconi, Gavin Fujimori, Boaz Odwar, Caitlin Cawley, Beth A. McCormick, Ann M. Moormann, Mireya Wessolossky, Vanni Bucci, and Ana Maldonado-Contreras

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Effects of COVID-19 on Health Care Workers, Mary Whalen and Penny C. Smith

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Season 2, Episode 2: Check Engine Light, Qiuwei Yang, Jesse Sardell, Heather Finlay-Morreale, and Hugh Silk

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Randomized controlled trial of sulforaphane and metabolite discovery in children with Autism Spectrum Disorder, Andrew W. Zimmerman, Kanwaljit Singh, Susan Connors, Hua Liu, Anita A. Panjwani, Li-Ching Lee, Eileen Diggins, Ann Foley, Stepan Melnyk, Indrapal N. Singh, S. Jill James, Richard E. Frye, and Jed W. Fahey

Publications from 2020

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Dipeptide repeat proteins inhibit homology-directed DNA double strand break repair in C9ORF72 ALS/FTD, Nadja S. Andrade, Abbas Abdallah, Christian Mueller, and Zane Zeier

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Sarcoid-like reaction in a patient recovering from coronavirus disease 19 pneumonia, Sara Behbahani, Julia Baltz, Rebecca Droms, April C. Deng, Shinya U. Amano, Nikki A. Levin, Mary Callery O'Brien, and Karen Wiss

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Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophy, Craig Campbell and Brenda L. Wong

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Adverse Childhood Experience and Social Risk: Pediatric Practice and Potential, Anne-Marie Conn, Moira Szilagyi, and Heather Forkey

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Silencing Trisomy 21 with XIST in Neural Stem Cells Promotes Neuronal Differentiation, Jan T. Czerminski and Jeanne B. Lawrence

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A Translational Model for Venous Thromboembolism: MicroRNA Expression in Hibernating Black Bears, Amanda Fazzalari, Giacomo Basadonna, Alper Kucukural, Kahraman Tanriverdi, Milka Koupenova-Zamor, Eric W. Dickson, Meghan Blackwood, Christian Mueller, Jane E. Freedman, and Mitchell A. Cahan

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Accelerated Graduation and the Deployment of New Physicians During the COVID-19 Pandemic, Terence R. Flotte, Anne C. Larkin, Melissa A. Fischer, Sonia N. Chimienti, Deborah M. DeMarco, Pang-Yen Fan, and Michael F. Collins

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Impact of Selective Eating on Social Domains Among Transition-Age Youth with Autism Spectrum Disorder: A Qualitative Study, Sara C. Folta, Carol Curtin, Aviva Must, Annie Pehrson, Katelyn Ryan, and Linda G. Bandini

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Muscle-Directed Delivery of an AAV1 Vector Leads to Capsid-Specific T Cell Exhaustion in Nonhuman Primates and Humans, Gwladys Gernoux, Alisha M. Gruntman, Meghan Blackwood, Marina Zieger, Terence R. Flotte, and Christian Mueller

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Prognostic factors for changes in the timed 4-stair climb in patients with Duchenne muscular dystrophy, and implications for measuring drug efficacy: A multi-institutional collaboration, Nathalie Goemans, Brenda L. Wong, Marleen Van den Hauwe, James Signorovitch, Gautam Sajeev, David Cox, John Landry, Madeline Jenkins, Ibrahima Dieye, Zhiwen Yao, Intekhab Hossain, and Susan J. Ward

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Impact of Intermittent Hypoxia on Growth in Very- and Extremely-Preterm Infants, Megan C. Horgan

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Children Exposed to Maltreatment: Assessment and the Role of Psychotropic Medication, Brooks Keeshin, Heather Forkey, George Fouras, and Harriet L. MacMillan

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Lentiviral gene therapy for X-linked chronic granulomatous disease, Donald B. Kohn, Peter E. Newburger, Claire Booth, Elizabeth M. Kang, Sung-Yun Pai, Kit L. Shaw, Giorgia Santilli, Myriam Armant, Karen F. Buckland, Uimook Choi, Suk See. De Ravin, Morna J. Dorsey, Diego Leon-Rico, and Christine Rivat

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Skin rash, strawberry tongue, and conjunctivitis in a teenage male, Nicholas Leonard, Karan Lal, Leah Belazarian, Christina Hermos, and Karen Wiss

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Biomarker Exploration in Human Peripheral Blood Mononuclear Cells for Monitoring Sulforaphane Treatment Responses in Autism Spectrum Disorder, Hua Liu, Andrew W. Zimmerman, Kanwaljit Singh, Susan Connors, Eileen Diggins, Katherine K. Stephenson, Albena T. Dinkova-Kostova, and Jed W. Fahey

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Real-world outcomes of long-term prednisone and deflazacort use in patients with Duchenne muscular dystrophy: experience at a single, large care center, Jessica R. Marden, Jonathan Freimark, Zhiwen Yao, James Signorovitch, Cuixia Tian, and Brenda L. Wong

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Cytomegalovirus-Specific T Cell Epitope Recognition in Congenital Cytomegalovirus Mother-Infant Pairs, Emma C. Materne, Daniele Lilleri, Francesca Garofoli, Giuseppina Lombardi, Milena Furione, Maurizio Zavattoni, and Laura L. Gibson

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COVID-19 in the Pediatric Population, Patricia A. McQuilkin

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Welcome to the PEER COVID-19 Seminar, Patricia A. McQuilkin

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Bone outcomes following sleeve gastrectomy in adolescents and young adults with obesity versus non-surgical controls, Madhusmita Misra, Vibha Singhal, Brian Carmine, Amita Bose, Megan M. Kelsey, Fatima Cody Stanford, Jennifer Bram, Jeremy T. Aidlen, Thomas Inge, Mary L. Bouxsein, and Miriam A. Bredella

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Association of Damaging Variants in Genes With Increased Cancer Risk Among Patients With Congenital Heart Disease, Sarah U. Morton, Peter E. Newburger, and Christine E. Seidman

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Pediatric Refugee Health Care Delivery in the Community Setting: An Educational Workshop for Multidisciplinary Family-Centered Care During Resettlement, Umbereen S. Nehal, Satoko Kanahara, Mihoko Tanabe, Grace Hayner, and Brett D. Nelson

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Mechanisms and early patterns of dyslipidemia in pediatric type 1 and type 2 diabetes, Benjamin U. Nwosu, Tony R. Villalobos-Ortiz, Gabrielle Jasmin, Sadichchha Parajuli, Emily Zitek-Morrison, and Bruce A. Barton

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Continuous glucose monitoring reduces pubertal hyperglycemia of type 1 diabetes, Benjamin U. Nwosu, Shamima Yeasmin, Sanaa S. Ayyoub, Shwetha Rupendu, Tony R. Villalobos-Ortiz, Gabrielle Jasmin, Sadichchha Parajuli, Bita Zahedi, Emily Zitek-Morrison, Laura C. Alonso, and Bruce A. Barton

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CK2-Dependent Phosphorylation of the Brg1 Chromatin Remodeling Enzyme Occurs during Mitosis, Teresita Padilla-Benavides, Dominic T. Haokip, Yeonsoo Yoon, Pablo Reyes-Gutierrez, Jaime A. Rivera, and Anthony N. Imbalzano

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Improved prime editors enable pathogenic allele correction and cancer modelling in adult mice [preprint], Pengpeng Liu, Shun-Qing Liang, Chunwei Zheng, Esther Mintzer, Yan G. Zhao, Karthikeyan Ponnienselvan, Aamir Mir, Erik J. Sontheimer, Guangping Gao, Terence R. Flotte, Scot A. Wolfe, and Wen Xue

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Characteristics and Outcomes of Children With Coronavirus Disease 2019 (COVID-19) Infection Admitted to US and Canadian Pediatric Intensive Care Units, Lara S. Shekerdemian, Nabihah R. Mahmood, Katie K. Wolfe, Becky J. Riggs, Catherine E. Ross, Christine A. McKiernan, Sabrina M. Heidemann, Lawrence C. Kleinman, Anita I. Sen, Mark W. Hall, Margaret A. Priestley, John K. McGuire, Konstantinos Boukas, Matthew P. Sharron, Jeffrey P. Burns, and International COVID-19 PICU Collaborative

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Duchenne Muscular Dystrophy: the Heart of the Matter, Jeffrey Shih, Alejandro Folch, and Brenda L. Wong

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Developmental regression and mitochondrial function in children with autism, Kanwaljit Singh, Indrapal N. Singh, Eileen Diggins, Susan Connors, Mohammad A. Karim, David Lee, Andrew W. Zimmerman, and Richard E. Frye

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Global Guide: A Comprehensive Global Health Education Resource for Pediatric Program Directors, Nicole E. St Clair, Alhassan Abdul-Mumin, Sumeet L. Banker, Tania Condurache, Heather Crouse, Heather Haq, Jill Helphinstine, Peter Nicholas Kazembe, Stephanie Marton, Patricia A. McQuilkin, Michael B. Pitt, Marideth Rus, Christiana M. Russ, Chuck Schubert, Gordon E. Schutze, and Global Health Task Force of the American Board of Pediatrics

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Branched-chain alpha-ketoacid dehydrogenase deficiency (maple syrup urine disease): Treatment, biomarkers, and outcomes, Kevin A. Strauss

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Glutaric acidemia type 1: Treatment and outcome of 168 patients over three decades, Kevin A. Strauss

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Volume and Infusion Rate Dynamics of Intraparenchymal Central Nervous System Infusion in a Large Animal Model, Toloo Taghian, Erin Horn, Mohammed S. Shazeeb, Lindsey J. Bierfeldt, Susan M. Tuominen, Jennifer Koehler, Deborah Fernau, Stephanie Bertrand, Stephen Frey, Oguz Cataltepe, Matthew J. Gounis, Aly Abayazeed, Terence R. Flotte, Miguel Sena-Esteves, and Heather Gray-Edwards

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Two-Plasmid Packaging System for Recombinant Adeno-Associated Virus, Qiushi Tang, Allison M. Keeler, Songbo Zhang, Qin Su, Zhuoyao Lyu, Yangfan Cheng, Guangping Gao, and Terence R. Flotte

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The care of patients with Duchenne, Becker and other muscular dystrophies in the COVID-19 pandemic, Aravindhan Veerapandiyan, Kathryn R. Wagner, Susan Apkon, Craig M. McDonald, Katherine D. Mathews, Julie A. Parsons, Brenda L. Wong, Katy Eichinger, Perry B. Shieh, Russell J. Butterfield, Vamshi K. Rao, Edward C. Smith, Crystal M. Proud, Anne M. Connolly, and Emma Ciafaloni

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Defining a new immune deficiency syndrome: MAN2B2-CDG, Jan Verheijen, Sunnie Y. Wong, Jared H. Rowe, Kimiyo Raymond, Jennifer Stoddard, Ottavia M. Delmonte, Marita Bosticardo, Kerry Dobbs, Julie Niemela, Enrica Calzoni, Sung-Yun Pai, Uimook Choi, Yasuhiro Yamazaki, Anne Marie Comeau, and Eva Morava

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Electronic Health Record Portal Messages and Interactive Voice Response Calls to Improve Rates of Early Season Influenza Vaccination: Randomized Controlled Trial, Jessica G. Wijesundara, Mayuko Ito Fukunaga, Jessica Ogarek, Bruce A. Barton, Lloyd D. Fisher, Peggy Preusse, Devi Sundaresan, Lawrence D. Garber, Kathleen M. Mazor, and Sarah L. Cutrona

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Online Learning and Residents' Acquisition of Mechanical Ventilation Knowledge: Sequencing Matters, Traci A. Wolbrink, Sandrijn M. van Schaik, David A. Turner, Steven J. Staffa, Eleanor Keller, Donald L. Boyer, Grace Chong, Jarrod Cross, Sylvia Del Castillo, Andrew Feng, R. Stanley Hum, Ebor Jacob James, Amanda Johnson, Sarah Kandil, and Martin Kneyber

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Predicting Mortality in Children With Pediatric Acute Respiratory Distress Syndrome: A Pediatric Acute Respiratory Distress Syndrome Incidence and Epidemiology Study, Nadir Yehya and Stacey L. Valentine

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Implantation and Gastrulation Abnormalities Characterize Early Embryonic Lethal Mouse Lines [preprint], Yeonsoo Yoon, Joy Riley, Judith Gallant, Ping Xu, and Jaime A. Rivera-Pérez

Publications from 2019

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What the Gene Therapy Community Should Do About Sexual Harassment, Jennifer E. Adair and Terence R. Flotte

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A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system, Julia F. Alterman, Bruno M. D. C. Godinho, Matthew R. Hassler, Chantal M. Ferguson, Dimas Echeverria, Ellen Sapp, Reka A. Haraszti, Andrew H. Coles, Faith Conroy, Rachael Miller, Loic Roux, Paul Yan, Emily G. Knox, Anton A. Turanov, Robert M. King, Gwladys Gernoux, Christian Mueller, Heather Gray-Edwards, Richard P. Moser, N Bishop, Samer M. Jaber, Matthew J. Gounis, Miguel Sena-Esteves, Athma A. Pai, Marian DiFiglia, Neil Aronin, and Anastasia Khvorova

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Benign ethnic neutropenia, Suheil Albert Atallah-Yunes, Audrey Ready, and Peter E. Newburger

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Special Considerations in the Systematic Psychosocial Screening of Youth with Type 1 Diabetes, Samantha A. Barry-Menkhaus, Alison M. Stoner, Kristin L. MacGregor, and Leslie A. Soyka

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New Horizons for Immune Gene Therapy, Adi Barzel and Terence R. Flotte

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Cannabinoid toxicity in pediatrics, Eike Blohm, Peter J. Sell, and Mark J. Neavyn

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Investigating Potential Biomarkers in Autism Spectrum Disorder, Carolyn Bridgemohan, David E. Cochran, Andrew W. Zimmerman, Roula N. Choueiri, and Jean A. Frazier

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Comparison of artifacts between paste and collodion method of electrode application in pediatric EEG, Danielle Brigham, Yash Shah, Kanwaljit Singh, Ivan Pavkovic, Shefali Karkare, and Sanjeev V. Kothare

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Presentation and Treatment Outcomes of Liberian Children Age 5 Years and Under Diagnosed With Severe Malaria, Benetta Collins-Andrews, Patricia A. McQuilkin, Kanagasabai Udhayashankar, Eric Adu, and Ann M. Moormann

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MicroRNAs Absent in Caput Sperm Are Required for Normal Embryonic Development, Colin C. Conine, Fengyun Sun, Lina Song, Jaime A. Rivera-Perez, and Oliver J. Rando

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MED20 is essential for early embryogenesis and regulates NANOG expression, Wei Cui, Chelsea Marcho, Yongsheng Wang, Rinat Degani, Morgane Golan, Kimberly D. Tremblay, Jaime A. Rivera-Pérez, and Jesse Mager

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A Compact, High-Accuracy Cas9 with a Dinucleotide PAM for In Vivo Genome Editing, Alireza Edraki, Aamir Mir, Raed Ibraheim, Ildar Gainetdinov, Yeonsoo Yoon, Chun-Qing Song, Yueying Cao, Judith Gallant, Wen Xue, Jaime A. Rivera, and Erik J. Sontheimer

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Senior Gene Therapy Scientists Take a Stand Against Human Embryo Gene Editing, Terence R. Flotte

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Top Five Gene Therapy Stories of 2019, Terence R. Flotte

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Putting Your Trauma Lens On, Heather Forkey

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Higher order genomic organization and epigenetic control maintain cellular identity and prevent breast cancer, A. J. Fritz, Jeffrey A. Nickerson, Anthony N. Imbalzano, and Gary S. Stein

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Neutropenia in the age of genetic testing: Advances and challenges, Elissa Furutani, Peter E. Newburger, and Akiko Shimamura

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Aberrant splicing contributes to severe alpha-spectrin-linked congenital hemolytic anemia, Patrick G. Gallagher and Peter E. Newburger

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Bridging from Intramuscular to Limb Perfusion Delivery of rAAV: Optimization in a Non-human Primate Study, Alisha Gruntman, Gwladys Gernoux, Qiushi Tang, Guo-Jie Ye, Dave R. Knop, Gensheng Wang, Janet Benson, Kristen E. Coleman, Allison M. Keeler, Christian Mueller, Louis G. Chicoine, Jeffrey D. Chulay, and Terence R. Flotte

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Lithium-Induced Chronic Kidney Disease in a Pediatric Patient, Neena Gupta, Meghan Gibson, and Ellen C. Wallace

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RUNX1-dependent mechanisms in biological control and dysregulation in cancer, Deli Hong, Andrew J. Fritz, Jonathan A. Gordon, Coralee E. Tye, Joseph R. Boyd, Kirsten M. Tracy, Seth E. Frietze, Frances E. Carr, Jeffrey A. Nickerson, Andre J. Van Wijnen, Anthony N. Imbalzano, Sayyed K. Zaidi, Jane B. Lian, Janet L. Stein, and Gary S. Stein

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Women's Representation Among Members and Leaders of National Medical Specialty Societies, Reshma Jagsi, Olivia Means, Diana Lautenberger, Rochelle D. Jones, Kent A. Griffith, Terence R. Flotte, Lynn K. Gordon, Kathryn M. Rexrode, Lori W. Wagner, and Archana Chatterjee

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Recombinant Adeno-Associated Virus Gene Therapy in Light of Luxturna (and Zolgensma and Glybera): Where Are We, and How Did We Get Here, Allison M. Keeler and Terence R. Flotte

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Intralingual and Intrapleural AAV Gene Therapy Prolongs Survival in a SOD1 ALS Mouse Model, Allison M. Keeler, Marina Zieger, Carson Semple, Logan Pucci, Alessandra Veinbachs, Robert H. Brown Jr., Christian Mueller, and Mai K. Elmallah