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 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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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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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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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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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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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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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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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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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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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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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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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

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

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Pollen-derived RNAs Are Found in the Human Circulation, Milka Koupenova-Zamor, Eric O. Mick, Heather A. Corkrey, Anupama Singh, Selim E. Tanriverdi, Olga Vitseva, Daniel Levy, Allison M. Keeler, Marzieh Ezzaty Mirhashemi, Mai K. Elmallah, Mark Gerstein, Joel Rozowsky, Kahraman Tanriverdi, and Jane E. Freedman

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Case 28-2019: A 22-Year-Old Woman with Dyspnea and Chest Pain, Ted M. Kremer, Mark E. Lindsay, T Bernard. Kinane, Megan H. Hawley, Brent P. Little, and Mari Mino-Kenudson

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Electrical impedance myography for reducing sample size in Duchenne muscular dystrophy trials, Melanie L. Leitner, Kush Kapur, Basil T. Darras, Michele Yang, Brenda Wong, Laura Dalle Pazze, Julaine Florence, Martin Buck, Laura Freedman, Jose Bohorquez, Seward Rutkove, and Craig Zaidman

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Pediatric Endoscopy and High-risk Patients: A Clinical Report From the NASPGHAN Endoscopy Committee, Jenifer R. Lightdale, Quin Y. Liu, Benjamin Sahn, David M. Troendle, Mike Thomson, and Douglas S. Fishman

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Reference Managers, Patricia A. McQuilkin

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BRG1 is a prognostic indicator and a potential therapeutic target for prostate cancer, Rohini Muthuswami, LeeAnn Bailey, Radhakrishnan Rakesh, Anthony N. Imbalzano, Jeffrey A. Nickerson, and Joel W. Hockensmith

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Partial Clinical Remission of Type 1 Diabetes Mellitus in Children: Clinical Applications and Challenges with its Definitions, Benjamin U. Nwosu

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Pubertal lipid levels are significantly lower in youth with type 1 diabetes who experienced partial clinical remission, Benjamin U. Nwosu, Shwetha Rupendu, Emily Zitek-Morrison, Deepa Patel, Tony R. Villalobos-Ortiz, Gabrielle Jasmin, and Bruce A. Barton

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Improving Efficiency and Communication around Sedated Fracture Reductions in a Pediatric Emergency Department, Niloufar Paydar-Darian, Michael P. Goldman, Kenneth A. Michelson, Katharine C. Button, Elizabeth K. Hewett, Theodore Macnow, Andrew F. Miller, Megan A. Musisca, Joel D. Hudgins, and Matthew A. Eisenberg

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Condylomata acuminata (anogenital warts) contain accumulations of HIV-1 target cells that may provide portals for HIV transmission, Jeffrey Pudney, Zoon Wangu, Lori Panther, Dana Fugelso, Jai Marathe, Manish Sagar, Joseph A. Politch, and Deborah J. Anderson

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Two Unique Cases of X-linked SCID: A Diagnostic Challenge in the Era of Newborn Screening, Pooja Purswani, Anne Marie Comeau, Jaime E. Hale, and Jolan E. Walter

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Preparing Medical Students to Be Physician Leaders: A Leadership Training Program for Students Designed and Led by Students, Kristen Richard, Michael G. Noujaim, Luanne A. Thorndyke, and Melissa A. Fischer

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Salivary cortisol levels as a biomarker for severity of withdrawal in opioid-exposed newborns, Nicolas Rodriguez, Mark Vining, and Elisabeth Bloch-Salisbury

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How we approach: Severe congenital neutropenia and myelofibrosis due to mutations in VPS45, Bella Shadur, Nathalie Asherie, Peter E. Newburger, and Polina Stepensky

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Genetic Deficiency of Flavin-Containing Monooxygenase 3 ( Fmo3) Protects Against Thrombosis but Has Only a Minor Effect on Plasma Lipid Levels-Brief Report, Diana M. Shih, Marina Zieger, Christian Mueller, and Aldons J. Lusis

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Age-and-sex stratified prevalence of atrial fibrillation in rural Western India: Results of SMART-India, a population-based screening study, Apurv Soni, Sunil Karna, Nisha Fahey, Saket Sanghai, Harshil Patel, Shyamsundar Raithatha, Sunil Thanvi, Somashekhar Nimbalkar, Ben Freedman, Jeroan J. Allison, and David D. McManus

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A Safe and Reliable Technique for CNS Delivery of AAV Vectors in the Cisterna Magna, Toloo Taghian, Miklos G. Marosfoi, Ajit S. Puri, Oguz Cataltepe, Robert M. King, Deborah Fernau, Ana Rita Batista, Terence R. Flotte, Diane McKenna-Yasek, Phillip W. L. Tai L, Neil Aronin, Matthew J. Gounis, Miguel Sena-Esteves, and Heather L. Gray-Edwards

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Asthma in Children and Adults-What Are the Differences and What Can They Tell us About Asthma, Michelle K. Trivedi and Eve Denton

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Alignment of stakeholder agendas to facilitate the adoption of school-supervised asthma therapy, Michelle Trivedi, Janki Patel, Shushmita Hoque, Raphael Mizrahi, Kathleen Biebel, Wanda Phipatanakul, Stephenie C. Lemon, Nancy Byatt, Lynn B. Gerald, Milagros C. Rosal, and Lori Pbert

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Circumventing cellular immunity by miR142-mediated regulation sufficiently supports rAAV-delivered OVA expression without activating humoral immunity, Yuanyuan Xiao, Manish Muhuri, Shaoyong Li, Guangchao Xu, Li Luo, Jia Li, Dan Wang, Qin Su, M. Abu Nahid, Christian Mueller, Phillip W. L. Tai, and Guangping Gao

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AAV9 gene replacement therapy for respiratory insufficiency in very-long chain acyl-CoA dehydrogenase deficiency, Marina Zieger, Allison M. Keeler, Terence R. Flotte, and Mai K. ElMallah

Publications from 2018

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TIP55, a splice isoform of the KAT5 acetyltransferase, is essential for developmental gene regulation and organogenesis, Diwash Acharya, Bernadette Nera, Zachary J. Milstone, Lauren Bourke, Yeonsoo Yoon, Jaime A. Rivera-Pérez, Chinmay M. Trivedi, and Thomas G. Fazzio

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Guidelines for sedation and anesthesia in GI endoscopy, ASGE Standards of Practice Committee, Dayna S. Early, Jenifer R. Lightdale, and John M. DeWitt

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A Child With an Unusual Retained Oral Foreign Body, Timothy Baloda, Megan L. McBurnie, and Theodore Macnow

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Recommendations on the Indications for RBC Transfusion for the Critically Ill Child Receiving Support From Extracorporeal Membrane Oxygenation, Ventricular Assist, and Renal Replacement Therapy Devices From the Pediatric Critical Care Transfusion and Anemia Expertise Initiative, Melania M. Bembea, Ira M. Cheifetz, James D. Fortenberry, Timothy E. Bunchman, Stacey L. Valentine, Scot T. Bateman, and Marie E. Steiner

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The Pediatric Critical Care Transfusion and Anemia Expertise Initiative Consensus Conference Methodology, Melania M. Bembea, Stacey L. Valentine, Scot T. Bateman, Lisa M. Wilson, Blair Anton, Nancy E. Harger, and Karen A. Robinson

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Safe and effective superoxide dismutase 1 silencing using artificial microRNA in macaques, Florie Borel, Gwladys Gernoux, Huaming Sun, Rachel Stock, Meghan Blackwood, Robert H. Brown Jr., and Christian Mueller

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Editing out five Serpina1 paralogs to create a mouse model of genetic emphysema, Florie Borel, Huaming Sun, Marina Zieger, Andrew Cox, Brynn Cardozo, Weiying Li, Gabriella Oliveira, Airiel Davis, Alisha Gruntman, Terence R. Flotte, Michael H. Brodsky, Andrew M. Hoffman, Mai K. Elmallah, and Christian Mueller

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Lack of acute xenogeneic graft- versus-host disease, but retention of T-cell function following engraftment of human peripheral blood mononuclear cells in NSG mice deficient in MHC class I and II expression, Michael A. Brehm, Laurie L. Kenney, Michael V. Wiles, Benjamin E. Low, Roland M. Tisch, Lisa Burzenski, Christian Mueller, Dale L. Greiner, and Leonard D. Shultz

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Expression of inflammatory markers in women with perinatal depressive symptoms, Rachel Buglione-Corbett, Kristina M. Deligiannidis, Katherine Leung, Ning Zhang, Mary M. Lee, Milagros C. Rosal, and Tiffany A. Moore Simas

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Meeting the Late-Career Needs of Faculty Transitioning Through Retirement: One Institution's Approach, Joanna Cain, Marianne E. Felice, Judith K. Ockene, Robert J. Milner, John L. Congdon, Stephen Tosi, and Luanne A. Thorndyke

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Trisomy silencing by XIST normalizes Down syndrome cell pathogenesis demonstrated for hematopoietic defects in vitro, Jen-Chieh Chiang, Jun Jiang, Peter E. Newburger, and Jeanne B. Lawrence

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Assuring Integrity in the Residency Match Process, Sonia N. Chimienti, Deborah M. DeMarco, Terence R. Flotte, and Michael F. Collins

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Recommendations on RBC Transfusion in Infants and Children With Acquired and Congenital Heart Disease From the Pediatric Critical Care Transfusion and Anemia Expertise Initiative, Jill M. Cholette, Ariane Willems, Stacey L. Valentine, Scot T. Bateman, and Steven M. Schwartz

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Small RNAs gained during epididymal transit of sperm are essential for embryonic development in mice, Colin C. Conine, Fengyun Sun, Lina Song, Jaime A. Rivera-Perez, and Oliver J. Rando

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Small RNAs Gained during Epididymal Transit of Sperm Are Essential for Embryonic Development in Mice, Colin C. Conine, Fengyun Sun, Lina Song, Jaime A. Rivera-Perez, and Oliver J. Rando

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Recommendations on RBC Transfusions in Critically Ill Children With Acute Respiratory Failure From the Pediatric Critical Care Transfusion and Anemia Expertise Initiative, Pierre Demaret, Guillaume Emeriaud, Nabil E. Hassan, Martin C. J. Kneyber, Stacey L. Valentine, Scot T. Bateman, and Marisa Tucci

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Recommendations on RBC Transfusion in General Critically Ill Children Based on Hemoglobin and/or Physiologic Thresholds From the Pediatric Critical Care Transfusion and Anemia Expertise Initiative, Allan Doctor, Jill M. Cholette, Kenneth E. Remy, Andrew Argent, Jeffrey L. Carson, Stacey L. Valentine, Scot T. Bateman, and Jacques Lacroix

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Airway Basal Cells Are the Key for Cystic Fibrosis Gene Therapy, Terence R. Flotte

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Recombinant Adeno-Associated Virus-Based Gene Therapy for Disorders Detected by Newborn Screening: Inherent Limitations of This Approach, Terence R. Flotte

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The Year in Review: The Top Five Papers of 2018, Terence R. Flotte

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Severe Toxicity in Nonhuman Primates and Piglets with Systemic High-Dose Administration of Adeno-Associated Virus Serotype 9-Like Vectors: Putting Patients First, Terence R. Flotte and Hildegard Buning

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Gene and Cell Therapy in China: Highlighting Excellence in the 21st Century 21, Terence R. Flotte and Guangping Gao

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Childhood Trauma Management in Primary Care, Heather Forkey and Anne-Marie Conn

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Gene expression in chronic granulomatous disease and interferon-gamma receptor-deficient cells treated in vitro with interferon-gamma, Josias B. Frazao, Martino Colombo, Cedric Simillion, Adem Bilican, Irene Keller, Daniel Wuthrich, Zhiqing Zhu, Michal J. Okoniewski, Remy Bruggmann, Antonio Condino-Neto, and Peter E. Newburger

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The clinical spectrum of pyruvate kinase deficiency: data from the Pyruvate Kinase Deficiency Natural History Study, Rachael F. Grace, Peter E. Newburger, and Wilma Barcellini

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Endocrine disrupters and pubertal timing, Louise C. Greenspan and Mary M. Lee

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The rapidly evolving state of gene therapy, Alisha Gruntman and Terence R. Flotte

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Differences in self-reported weekend catch up sleep between children and adolescents with and without primary hypertension, Neena Gupta, Louise Maranda, and Rakesh Gupta

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Self-Reported Management of IV Fluids and Fluid Accumulation in Children With Acute Respiratory Failure, Amanda B. Hassinger and Stacey L. Valentine