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.
Publications from 2020
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
Publications from 2019
What the Gene Therapy Community Should Do About Sexual Harassment, Jennifer E. Adair and Terence R. Flotte
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
Benign ethnic neutropenia, Suheil Albert Atallah-Yunes, Audrey Ready, and Peter E. Newburger
New Horizons for Immune Gene Therapy, Adi Barzel and Terence R. Flotte
Cannabinoid toxicity in pediatrics, Eike Blohm, Peter J. Sell, and Mark J. Neavyn
Investigating Potential Biomarkers in Autism Spectrum Disorder, Carolyn Bridgemohan, David E. Cochran, Andrew W. Zimmerman, Roula N. Choueiri, and Jean A. Frazier
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
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
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
Senior Gene Therapy Scientists Take a Stand Against Human Embryo Gene Editing, Terence R. Flotte
Top Five Gene Therapy Stories of 2019, Terence R. Flotte
Putting Your Trauma Lens On, Heather Forkey
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
Neutropenia in the age of genetic testing: Advances and challenges, Elissa Furutani, Peter E. Newburger, and Akiko Shimamura
Aberrant splicing contributes to severe alpha-spectrin-linked congenital hemolytic anemia, Patrick G. Gallagher and Peter E. Newburger
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
Lithium-Induced Chronic Kidney Disease in a Pediatric Patient, Neena Gupta, Meghan Gibson, and Ellen C. Wallace
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
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
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
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
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
Partial Clinical Remission of Type 1 Diabetes Mellitus in Children: Clinical Applications and Challenges with its Definitions, Benjamin U. Nwosu
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
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
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
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
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
How we approach: Severe congenital neutropenia and myelofibrosis due to mutations in VPS45, Bella Shadur, Nathalie Asherie, Peter E. Newburger, and Polina Stepensky
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
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
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
Asthma in Children and Adults-What Are the Differences and What Can They Tell us About Asthma, Michelle K. Trivedi and Eve Denton
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
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
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
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
Guidelines for sedation and anesthesia in GI endoscopy, ASGE Standards of Practice Committee, Dayna S. Early, Jenifer R. Lightdale, and John M. DeWitt
A Child With an Unusual Retained Oral Foreign Body, Timothy Baloda, Megan L. McBurnie, and Theodore Macnow
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
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
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
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
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
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
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
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
Assuring Integrity in the Residency Match Process, Sonia N. Chimienti, Deborah M. DeMarco, Terence R. Flotte, and Michael F. Collins
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
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
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
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
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
Airway Basal Cells Are the Key for Cystic Fibrosis Gene Therapy, Terence R. Flotte
Recombinant Adeno-Associated Virus-Based Gene Therapy for Disorders Detected by Newborn Screening: Inherent Limitations of This Approach, Terence R. Flotte
The Year in Review: The Top Five Papers of 2018, Terence R. Flotte
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
Gene and Cell Therapy in China: Highlighting Excellence in the 21st Century 21, Terence R. Flotte and Guangping Gao
Childhood Trauma Management in Primary Care, Heather Forkey and Anne-Marie Conn
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
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
Endocrine disrupters and pubertal timing, Louise C. Greenspan and Mary M. Lee
The rapidly evolving state of gene therapy, Alisha Gruntman and Terence R. Flotte
Differences in self-reported weekend catch up sleep between children and adolescents with and without primary hypertension, Neena Gupta, Louise Maranda, and Rakesh Gupta
Self-Reported Management of IV Fluids and Fluid Accumulation in Children With Acute Respiratory Failure, Amanda B. Hassinger and Stacey L. Valentine
Inhibition of protein arginine methyltransferase 5 enhances hepatic mitochondrial biogenesis, Lei Huang, Jehnan Liu, Xiao-Ou Zhang, Katelyn Sibley, Sonia M. Najjar, Mary M. Lee, and Joae Qiong Wu
A Community-Based Pilot Survey of Pediatric Food Allergies in a Maryland Orthodox Jewish Community, Peter A. Kahn, Michelle K. Trivedi, Amira A. Ramadan, William J. Sheehan, Carter R. Petty, and Wanda Phipatanakul
Recommendations on RBC Transfusion in Critically Ill Children With Nonlife-Threatening Bleeding or Hemorrhagic Shock From the Pediatric Critical Care Transfusion and Anemia Expertise Initiative, Oliver Karam, Robert T. Russell, Paul Stricker, Adam M. Vogel, Scot T. Bateman, Stacey L. Valentine, and Philip C. Spinella
Systemic Delivery of AAVB1-GAA Clears Glycogen and Prolongs Survival in a Mouse Model of Pompe Disease, Allison M. Keeler, Marina Zieger, Sophia H. Todeasa, Angela L. McCall, Jennifer C. Gifford, Samantha Birsak, Sourav Roy Choudhury, Barry J. Byrne, Miguel Sena-Esteves, and Mai K. Elmallah
Single-Center Experience With Liver Transplant Using Donors With Very High Transaminase Levels, Paulo N.A. Martins, Amanda Rawson, Babak Movahedi, Isabel M. A. Bruggenwirth, Natasha H. Dolgin, Ann-Britt Martins, Paria Mahboub, and Adel Bozorgzadeh
Literature Search Handout, Patricia A. McQuilkin
Workshop I: Research Training Wrap-up, Patricia A. McQuilkin, Steven C. Hatch, and Troy Moon
Recommendations on RBC Transfusions for Critically Ill Children With Nonhemorrhagic Shock From the Pediatric Critical Care Transfusion and Anemia Expertise Initiative, Jennifer A. Muszynski, Nina A. Guzzetta, Mark W. Hall, Duncan Macrae, Stacey L. Valentine, Scot T. Bateman, and Philip C. Spinella
"How I approach..."- A new series in Pediatric Blood and Cancer, Peter E. Newburger and Thomas G. Gross
Comment on Redondo et al. Racial/Ethnic Minority Youth With Recent-Onset Type 1 Diabetes Have Poor Prognostic Factors. Diabetes Care 2018;41:1017-1024, Benjamin U. Nwosu
Data from: Tobacco smoke exposure is an independent predictor of vitamin D deficiency in US children, Benjamin U. Nwosu and Philip Kum-Nji
Tobacco smoke exposure is an independent predictor of vitamin D deficiency in US children, Benjamin U. Nwosu and Philip Kum-Nji
Children with type 1 diabetes who experienced a honeymoon phase had significantly lower LDL cholesterol 5 years after diagnosis, Benjamin U. Nwosu, Bo Zhang, Sanaa S. Ayyoub, Stephanie Choi, Tony R. Villalobos-Ortiz, Laura C. Alonso, and Bruce A. Barton
Data from: Children with type 1 diabetes who experienced a honeymoon phase had significantly lower LDL cholesterol 5 years after diagnosis, Benjamin U. Nwosu, Bo Zhang, Sanaa S. Ayyoub, Stephanie Choi, Tony R. Villalobos-Ortiz, Laura C. Alonso, and Bruce A. Barton
Delayed Treatment Acceleration in Patients with Rheumatoid Arthritis Who Have Inadequate Response to Initial Tumor Necrosis Factor Inhibitors: Data from the Corrona Registry, Dimitrios A. Pappas, Robert A. Gerber, Heather J. Litman, David Gruben, Jamie Geier, Winnie D. Hua, Connie Chen, YouFu Li, Joel M. Kremer, John S. Andrews, and Jeffrey A. Bourret
Results at 5 Years After Gene Therapy for RPE65-Deficient Retinal Dystrophy, Mark E. Pennesi, Richard G. Weleber, Paul Yang, Chris Whitebirch, Beverly Thean, Terence R. Flotte, Margaret Humphries, Elvira Chegarnov, Kathleen N. Beasley, J. Timothy Stout, and Jeffrey D. Chulay
Aerobic Glycolysis Is Essential for Normal Rod Function and Controls Secondary Cone Death in Retinitis Pigmentosa, Lolita Petit, Shan Ma, Joris Cipi, Shun-Yun Cheng, Marina Zieger, Nissim Hay, and Claudio Punzo
Aerobic Glycolysis Is Essential for Normal Rod Function and Controls Secondary Cone Death in Retinitis Pigmentosa, Lolita Petit, Shan Ma, Joris Cipi, Shun-Yun Cheng, Marina Zieger, Nissim Hay, and Claudio Punzo
Peripubertal serum dioxin concentrations and subsequent sperm methylome profiles of young Russian adults, J. Richard Pilsner, Mary M. Lee, Evgeny Rogaev, and Oleg Sergeyev
The optimization of home oxygen weaning in premature infants trial: Design, rationale, methods, and lessons learned, Alexander Procaskey, Heather White, Tregony Simoneau, Nadav Traeger, Thomas Lahiri, Elie G. Abu Jawdeh, Ted M. Kremer, Catherine Sheils, Kathleen Meyer, Ted Rosenkrantz, Sankaran Krishnan, Tyler Hartman, Henry Feldman, and Lawrence M. Rhein
Clinical and economic burden of procedural sedation-related adverse events and their outcomes: analysis from five countries, Rhodri Saunders, Jason A. Davis, Peter Kranke, Rachel Weissbrod, David K. Whitaker, and Jenifer R. Lightdale
Vomiting in Children, T. Matthew Shields and Jenifer R. Lightdale
Prevalence and characteristics of sleep-disordered breathing in familial dysautonomia, Kanwaljit Singh, Jose-Alberto Palma, Horacio Kaufmann, Nataliya Tkachenko, Lucy Norcliffe-Kaufmann, Christy Spalink, Mikhail Kazachkov, and Sanjeev V. Kothare
CHD3 helicase domain mutations cause a neurodevelopmental syndrome with macrocephaly and impaired speech and language, Lot Snijders Blok, Inderneel Sahai, and Philippe M. Campeau
In vivo genome editing partially restores alpha1-antitrypsin in a murine model of AAT deficiency, Chun-Qing Song, Dan Wang, Tingting Jiang, Kevin O'Connor, Qiushi Tang, Lingling Cai, Xiangrui Li, Zhiping Weng, Hao Yin, Guangping Gao, Christian Mueller, Terence R. Flotte, and Wen Xue
Case Definitions for Conditions Identified by Newborn Screening Public Health Surveillance, Marci K. Sontag, Deboshree Sarkar, Anne Marie Comeau, Kathryn Hassell, Lorenzo D. Botto, Richard Parad, Susan R. Rose, Kupper A. Wintergerst, Kim Smith-Whitley, Sikha Singh, Careema Yusuf, Jelili Ojodu, Sara Copeland, and Cynthia F. Hinton
Implementation of the Recommendations for RBC Transfusions for Critically Ill Children From the Pediatric Critical Care Transfusion and Anemia Expertise Initiative, Katherine M. Steffen, Scot T. Bateman, Stacey L. Valentine, Sara Small, Philip C. Spinella, and Allan Doctor
Recommendations on RBC Transfusion Support in Children With Hematologic and Oncologic Diagnoses From the Pediatric Critical Care Transfusion and Anemia Expertise Initiative, Marie E. Steiner, Nicole D. Zantek, Simon J. Stanworth, Robert I. Parker, Stacey L. Valentine, Leslie E. Lehmann, Cassandra D. Josephson, Scot T. Bateman, and Naomi L. C. Luban
Serum anti-flagellin and anti-lipopolysaccharide immunoglobulins as predictors of linear growth faltering in Pakistani infants at risk for environmental enteric dysfunction, Sana Syed, Najeeha T. Iqbal, Kamran Sadiq, Jennie Z. Ma, Tauseef Akhund, Wenjun Xin, Sean R. Moore, Enju Liu, Shahida Qureshi, Kerri B. Gosselin, Andrew Gewirtz, Christopher P. Duggan, and S Asad. Ali
The Genetic Landscape of Diamond-Blackfan Anemia, Jacob C. Ulirsch, Peter E. Newburger, Andrei A. Korostelev, Vijay G. Sankaran, and Hanna T. Gazda
Consensus Recommendations for RBC Transfusion Practice in Critically Ill Children From the Pediatric Critical Care Transfusion and Anemia Expertise Initiative, Stacey L. Valentine and Scot T. Bateman
Prevalence and management of iron overload in pyruvate kinase deficiency: report from the Pyruvate Kinase Deficiency Natural History Study, Eduard J. van Beers, Peter E. Newburger, and Rachael F. Grace
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, and Guangping Gao
Adeno-Associated Virus Neutralizing Antibodies in Large Animals and Their Impact on Brain Intraparenchymal Gene Transfer, Dan Wang, Li Zhong, Mengxin Li, Jia Li, Karen Tran, Lingzhi Ren, Ran He, Jun Xie, Richard P. Moser, Cara Fraser, Tim Kuchel, Miguel Sena-Esteves, Terence R. Flotte, Neil Aronin, and Guangping Gao