The Rokita Lab

Publications

Below, you’ll find a selection of our recent publications that highlight our contributions to pediatric oncology research and precision medicine. These papers showcase our commitment to advancing knowledge, developing innovative tools, and collaborating with the broader scientific community. Dive in to explore the insights we’ve gained and the impact we’re making!

Publications by Year

2026

Pan-pediatric brain tumor microRNA networks define immune microenvironment states and responses to CAR T-cell therapy
Kaleem Coleman, Ryan J Corbett, Zhongzhen Yi, Bicna Song, Alex Sickler, Aylar Babaei, Jo Lynne Rokita, Dalia Haydar
openRxiv  ·  23 Oct 2026  ·  doi:10.64898/2026.10.21.760687
Human leucocyte antigen class I and II alleles are associated with tumor susceptibility in neurofibromatosis type 1
Jonathan H. Sussman, Stephanie N. Brosius, Bernat Gel, Peiyao Li, Alvin Farrel, …, Eduard Serra, Kai Tan, Michael J. Fisher, John M. Maris, Thomas De Raedt
Frontiers in Immunology  ·  19 Oct 2026  ·  doi:10.3389/fimmu.2026.1966259
MOSAIC: Learning Graph Node Embeddings from Spectrally Isolated Dominant Subspaces of Accumulated Diffusion Operators.
Taha Mohseni Ahooyi, Benjamin J Stear, Yuanchao Zhang, Aditya Lahiri, James Terry, …, Christopher Nemarich, Jo Lynne Rokita, Sharon Diskin, Jonathan C Silverstein, Deanne M Taylor
bioRxiv : the preprint server for biology  ·  06 Oct 2026  ·  pubmed:42926505
Proteogenomic analysis of pediatric and AYA high-grade glioma reveals age-dependent biology, female-male differences, and kinase targets.
Nicole L Tignor, Mateusz Koptyra, Shrabanti Chowdhury, Francesca Petralia, Marina A Gritsenko, …, Amanda G Paulovich, Tao Liu, Adam Resnick, Brian R Rood, Pei Wang
Cell reports. Medicine  ·  17 Sep 2026  ·  pubmed:42753723
Tumor Microenvironmental Regulation of CAR T-Cell Therapy in High Risk Medulloblastoma.
Serge Yaacoub, Mostafa Seblani, Kaleem Coleman, Zhongzhen Yi, Kyoungtea Kim, …, Timothy Gershon, Jo Lynne Rokita, Martine F Roussel, Marina Sokolsky-Papkov, Dalia Haydar
Research square  ·  21 May 2026  ·  pubmed:42239754
Alternative splicing in pediatric central nervous system tumors highlights oncofetal candidate
Ammar S Naqvi, Patricia J Sullivan, Ryan J Corbett, Priyanka Sehgal, Karina L Conkrite, …, Peter J Madsen, Sharon J Diskin, Andrei Thomas-Tikhonenko, Adam C Resnick, Jo Lynne Rokita
Neuro-oncology pediatrics  ·  16 Feb 2026  ·  pubmed:41908886
Alternative splicing in pediatric central nervous system tumors highlights oncofetal candidate CLK1 exon 4

2025

Germline pathogenic variation impacts somatic alterations and patient outcomes in pediatric central nervous system tumors.
Ryan J Corbett, Rebecca S Kaufman, Shelly W McQuaid, Zalman Vaksman, Saksham Phul, …, Angela J Waanders, Miriam Bornhorst, Suzanne P MacFarland, Jo Lynne Rokita, Sharon J Diskin
Nature communications  ·  21 Nov 2025  ·  pubmed:41271695
Germline analysis of an international cohort of pediatric diffuse midline glioma patients.
Marion K Mateos, Pamela Ajuyah, Noemi Fuentes-Bolanos, Sam El-Kamand, Paulette Barahona, …, Sebastien Perreault, Chris Jones, Marie Wong-Erasmus, Mark J Cowley, David S Ziegler
Neuro-oncology  ·  08 Sep 2025  ·  pubmed:40072012
Kinome Reprogramming of G2/M Kinases and Repression of MYCN Contribute to Superior Efficacy of Lorlatinib in ALK-Driven Neuroblastoma.
Smita Matkar, Michael P East, Timothy J Stuhlmiller, Gabriela M Witek, Alvin Farrel, …, Jo Lynne Rokita, Mark Gerelus, John M Maris, Gary L Johnson, Yael P Mossé
Molecular cancer therapeutics  ·  02 Sep 2025  ·  pubmed:39907037
NRCAM variant defined by microexon skipping is a targetable cell surface proteoform in high-grade gliomas.
Priyanka Sehgal, Ammar S Naqvi, Makenna Higgins, Jiageng Liu, Kyra Harvey, …, Adam C Resnick, Hagen U Tilgner, Thomas De Raedt, Daniel J Powell, Andrei Thomas-Tikhonenko
Cell reports  ·  07 Aug 2025  ·  pubmed:40782352
Imaging Clusters of Pediatric Low-Grade Glioma Are Associated with Distinct Molecular Characteristics.
Anahita Fathi Kazerooni, Adam Kraya, Komal S Rathi, Meen Chul Kim, Varun Kesherwani, …, Sabine Mueller, Jo Lynne Rokita, Michael J Fisher, Adam C Resnick, Ali Nabavizadeh
AJNR. American journal of neuroradiology  ·  01 Aug 2025  ·  pubmed:39952665
Genetic ancestry superpopulations show distinct prevalence and outcomes across pediatric central nervous system tumors from the Pediatric Brain Tumor Atlas and Pediatric Neuro-Oncology Consortium.
Ryan J Corbett, Cricket C Gullickson, Zhuangzhuang Geng, Miguel A Brown, Bo Zhang, …, Angela J Waanders, Sebastian M Waszak, Sabine Mueller, Jo Lynne Rokita, Cassie Kline
Neuro-oncology  ·  21 Jun 2025  ·  pubmed:39847453
Germline Pathogenic DROSHA Variants Are Linked to Pineoblastoma and Wilms Tumor Predisposition.
Peter N Fiorica, Lisa Golmard, Jung Kim, Riyue Bao, Frank Y Lin, …, Douglas R Stewart, D Williams Parsons, Sharon E Plon, Kris Ann P Schultz, Kenan Onel
Clinical cancer research : an official journal of the American Association for Cancer Research  ·  14 Apr 2025  ·  pubmed:39992227
The Open Pediatric Cancer Project
Zhuangzhuang Geng, Eric Wafula, Ryan J Corbett, Yuanchao Zhang, Run Jin, …, Chuwei Zhong, Joseph M Dybas, Pei Wang, Deanne Taylor, Jo Lynne Rokita
GigaScience  ·  01 Jan 2025  ·  doi:10.1093/gigascience/giaf093
The Open Pediatric Cancer Project

2024

A proteogenomic surfaceome study identifies DLK1 as an immunotherapeutic target in neuroblastoma.
Amber K Hamilton, Alexander B Radaoui, Matthew Tsang, Daniel Martinez, Karina L Conkrite, …, Patrick H van Berkel, Malcolm A Smith, Benjamin A Garcia, John M Maris, Sharon J Diskin
Cancer cell  ·  24 Oct 2024  ·  pubmed:39454577
AutoGVP: a dockerized workflow integrating ClinVar and InterVar germline sequence variant classification.
Jung Kim, Ammar S Naqvi, Ryan J Corbett, Rebecca S Kaufman, Zalman Vaksman, …, Phillip B Storm, Adam C Resnick, Douglas R Stewart, Jo Lynne Rokita, Sharon J Diskin
Bioinformatics (Oxford, England)  ·  29 Feb 2024  ·  pubmed:38426335
AutoGVP
BARD1 germline variants induce haploinsufficiency and DNA repair defects in neuroblastoma.
Michael P Randall, Laura E Egolf, Zalman Vaksman, Minu Samanta, Matthew Tsang, …, Mehdi Layeghifard, Adam Shlien, John M Maris, Sharon J Diskin, Kristopher R Bosse
Journal of the National Cancer Institute  ·  10 Jan 2024  ·  pubmed:37688570

2023

Everolimus for Children With Recurrent or Progressive Low-Grade Glioma: Results From the Phase II PNOC001 Trial.
Daphne A Haas-Kogan, Mariam S Aboian, Jane E Minturn, Sarah E S Leary, Mohamed S Abdelbaki, …, Joanna J Phillips, Michael Prados, Annette M Molinaro, Sebastian M Waszak, Sabine Mueller
Journal of clinical oncology : official journal of the American Society of Clinical Oncology  ·  17 Nov 2023  ·  pubmed:37978951
Alternative lengthening of telomeres (ALT) in pediatric high-grade gliomas can occur without ATRX mutation and is enriched in patients with pathogenic germline mismatch repair (MMR) variants.
Jennifer L Stundon, Heba Ijaz, Krutika S Gaonkar, Rebecca S Kaufman, Run Jin, …, Suzanne P MacFarland, Douglas R Stewart, Sharon J Diskin, Jo Lynne Rokita, Kristina A Cole
Neuro-oncology  ·  06 Jul 2023  ·  pubmed:36541551
OpenPBTA: The Open Pediatric Brain Tumor Atlas.
Joshua A Shapiro, Krutika S Gaonkar, Stephanie J Spielman, Candace L Savonen, Chante J Bethell, …, Miguel A Brown, Adam C Resnick, Casey S Greene, Jo Lynne Rokita, Jaclyn N Taroni
Cell genomics  ·  31 May 2023  ·  pubmed:37492101
Identification of candidate genes for nicotine withdrawal in C57BL/6J × DBA/2J recombinant inbred mice
Maren L. Smith, Kristin M. Mignogna, Jo L. Rokita, Lorna MacLeod, M. Imad Damaj, Michael F. Miles
Genes, Brain and Behavior  ·  13 Feb 2023  ·  doi:10.1111/gbb.12844
Identification of candidate genes for nicotine withdrawal in C57BL/6J × DBA/2J recombinant inbred mice.
Maren L Smith, Kristin M Mignogna, Jo L Rokita, Lorna MacLeod, M Imad Damaj, Michael F Miles
Genes, brain, and behavior  ·  13 Feb 2023  ·  pubmed:36781202
Enhancing childhood cancer targetability.
Ammar S Naqvi, Jo Lynne Rokita
Nature cancer  ·  01 Feb 2023  ·  pubmed:36585448

2022

The children's brain tumor network (CBTN) - Accelerating research in pediatric central nervous system tumors through collaboration and open science.
Jena V Lilly, Jo Lynne Rokita, Jennifer L Mason, Tatiana Patton, Stephanie Stefankiewiz, …, Phillip B Storm, Rishi R Lulla, Michael Prados, Adam C Resnick, Angela J Waanders
Neoplasia (New York, N.Y.)  ·  03 Nov 2022  ·  pubmed:36335802
Upfront Biology-Guided Therapy in Diffuse Intrinsic Pontine Glioma: Therapeutic, Molecular, and Biomarker Outcomes from PNOC003.
Cassie Kline, Payal Jain, Lindsay Kilburn, Erin R Bonner, Nalin Gupta, …, Michael Prados, Adam Resnick, Sebastian M Waszak, Javad Nazarian, Sabine Mueller
Clinical cancer research : an official journal of the American Association for Cancer Research  ·  15 Sep 2022  ·  pubmed:35852795
Development of GPC2-directed chimeric antigen receptors using mRNA for pediatric brain tumors.
Jessica B Foster, Crystal Griffin, Jo Lynne Rokita, Allison Stern, Cameron Brimley, …, Phillip B Storm, David M Barrett, Adam C Resnick, John M Maris, Kristopher R Bosse
Journal for immunotherapy of cancer  ·  01 Sep 2022  ·  pubmed:36167467

2021

Macrophages in SHH subgroup medulloblastoma display dynamic heterogeneity that varies with treatment modality.
Mai T Dang, Michael V Gonzalez, Krutika S Gaonkar, Komal S Rathi, Patricia Young, …, Candace Savonen, Fernanda Mafra, Hakon Hakonarson, Tom Curran, Malay Haldar
Cell reports  ·  30 Mar 2021  ·  pubmed:33789113
GAS7 Deficiency Promotes Metastasis in MYCN-Driven Neuroblastoma.
Zhiwei Dong, Kok Siong Yeo, Gonzalo Lopez, Cheng Zhang, Erin N Dankert Eggum, …, Jan M van Deursen, Hu Li, Jennifer J Westendorf, John M Maris, Shizhen Zhu
Cancer research  ·  18 Feb 2021  ·  pubmed:33602789

2020

annoFuse: an R Package to annotate, prioritize, and interactively explore putative oncogenic RNA fusions.
Krutika S Gaonkar, Federico Marini, Komal S Rathi, Payal Jain, Yuankun Zhu, …, Pichai Raman, Adam C Resnick, Konstantin Strauch, Jaclyn N Taroni, Jo Lynne Rokita
BMC bioinformatics  ·  14 Dec 2020  ·  pubmed:33317447
Integrated Proteogenomic Characterization across Major Histological Types of Pediatric Brain Cancer.
Francesca Petralia, Nicole Tignor, Boris Reva, Mateusz Koptyra, Shrabanti Chowdhury, …, Amanda G Paulovich, Adam C Resnick, Phillip B Storm, Brian R Rood, Pei Wang
Cell  ·  25 Nov 2020  ·  pubmed:33242424
A transcriptome-based classifier to determine molecular subtypes in medulloblastoma.
Komal S Rathi, Sherjeel Arif, Mateusz Koptyra, Ammar S Naqvi, Deanne M Taylor, Phillip B Storm, Adam C Resnick, Jo Lynne Rokita, Pichai Raman
PLoS computational biology  ·  29 Oct 2020  ·  pubmed:33119584
LIN28B promotes neuroblastoma metastasis and regulates PDZ binding kinase.
Dongdong Chen, Julie Cox, Jayabhargav Annam, Melanie Weingart, Grace Essien, …, Daisy Li, Cara Shields, Oskar Laur, John M Maris, Robert W Schnepp
Neoplasia (New York, N.Y.)  ·  24 Apr 2020  ·  pubmed:32339949
Limited antitumor activity of combined BET and MEK inhibition in neuroblastoma.
Jason R Healy, Lori S Hart, Alexander L Shazad, Maria E Gagliardi, Matthew Tsang, …, Olena Barbash, Vandana Batra, Minu Samanta, John M Maris, Robert W Schnepp
Pediatric blood & cancer  ·  19 Apr 2020  ·  pubmed:32307821
Epigenomic profiling of neuroblastoma cell lines.
Kristen Upton, Apexa Modi, Khushbu Patel, Nathan M Kendsersky, Karina L Conkrite, …, Gregory I Sacks, Paolo Fortina, Sharon J Diskin, John M Maris, Jo Lynne Rokita
Scientific data  ·  14 Apr 2020  ·  pubmed:32286315
Telomere Maintenance Mechanisms Define Clinical Outcome in High-Risk Neuroblastoma.
Balakrishna Koneru, Gonzalo Lopez, Ahsan Farooqi, Karina L Conkrite, Thinh H Nguyen, …, Shengping Yang, David A Wheeler, John M Maris, Sharon J Diskin, C Patrick Reynolds
Cancer research  ·  14 Apr 2020  ·  pubmed:32291317
CAMKV Is a Candidate Immunotherapeutic Target in
Robyn T Sussman, Jo Lynne Rokita, Kevin Huang, Pichai Raman, Komal S Rathi, …, Maria Lane, Lori S Hart, Tricia Bhatti, Bruce Pawel, John M Maris
Frontiers in oncology  ·  06 Mar 2020  ·  pubmed:32211329
Pediatric high-grade glioma resources from the Children's Brain Tumor Tissue Consortium.
Heba Ijaz, Mateusz Koptyra, Krutika S Gaonkar, Jo Lynne Rokita, Valerie P Baubet, …, Maria Rita Santi, Phillip B Storm, Adam Resnick, Angela J Waanders, Kristina A Cole
Neuro-oncology  ·  11 Jan 2020  ·  pubmed:31613963

2019

ASCL1 is a MYCN- and LMO1-dependent member of the adrenergic neuroblastoma core regulatory circuitry.
Lu Wang, Tze King Tan, Adam D Durbin, Mark W Zimmerman, Brian J Abraham, …, Jo Lynne Rokita, John M Maris, Richard A Young, A Thomas Look, Takaomi Sanda
Nature communications  ·  09 Dec 2019  ·  pubmed:31819055
Combined innate and adaptive immunotherapy overcomes resistance of immunologically cold syngeneic murine neuroblastoma to checkpoint inhibition.
Julie Voeller, Amy K Erbe, Jacob Slowinski, Kayla Rasmussen, Peter M Carlson, …, John Maris, Jacquelyn A Hank, Zachary S Morris, Alexander L Rakhmilevich, Paul M Sondel
Journal for immunotherapy of cancer  ·  06 Dec 2019  ·  pubmed:31810498
Genomic Profiling of Childhood Tumor Patient-Derived Xenograft Models to Enable Rational Clinical Trial Design.
Jo Lynne Rokita, Komal S Rathi, Maria F Cardenas, Kristen A Upton, Joy Jayaseelan, …, Malcolm A Smith, Richard B Lock, Pichai Raman, David A Wheeler, John M Maris
Cell reports  ·  05 Nov 2019  ·  pubmed:31693904
YAP1 Mediates Resistance to MEK1/2 Inhibition in Neuroblastomas with Hyperactivated RAS Signaling.
Grace E Coggins, Alvin Farrel, Komal S Rathi, Colin M Hayes, Laura Scolaro, Jo Lynne Rokita, John M Maris
Cancer research  ·  31 Oct 2019  ·  pubmed:31672841
Germline 16p11.2 Microdeletion Predisposes to Neuroblastoma.
Laura E Egolf, Zalman Vaksman, Gonzalo Lopez, Jo Lynne Rokita, Apexa Modi, Patricia V Basta, Hakon Hakonarson, Andrew F Olshan, Sharon J Diskin
American journal of human genetics  ·  29 Aug 2019  ·  pubmed:31474320
The Pediatric Cell Atlas: Defining the Growth Phase of Human Development at Single-Cell Resolution.
Deanne M Taylor, Bruce J Aronow, Kai Tan, Kathrin Bernt, Nathan Salomonis, …, Yi Xing, Sek Won Kong, Carsten G Bönnemann, Kenneth D Mandl, Peter S White
Developmental cell  ·  28 Mar 2019  ·  pubmed:30930166

2018

Cross-Cohort Analysis Identifies a TEAD4-MYCN Positive Feedback Loop as the Core Regulatory Element of High-Risk Neuroblastoma.
Presha Rajbhandari, Gonzalo Lopez, Claudia Capdevila, Beatrice Salvatori, Jiyang Yu, …, Antonio Iavarone, Anna Lasorella, Jose M Silva, John M Maris, Andrea Califano
Cancer discovery  ·  06 Mar 2018  ·  pubmed:29510988

2017

Identification of GPC2 as an Oncoprotein and Candidate Immunotherapeutic Target in High-Risk Neuroblastoma.
Kristopher R Bosse, Pichai Raman, Zhongyu Zhu, Maria Lane, Daniel Martinez, …, Marco A Marra, Michael D Taylor, Dimiter S Dimitrov, Crystal L Mackall, John M Maris
Cancer cell  ·  11 Sep 2017  ·  pubmed:28898695
Transcriptomic profiling of 39 commonly-used neuroblastoma cell lines.
Jo Lynne Harenza, Maura A Diamond, Rebecca N Adams, Michael M Song, Heather L Davidson, Lori S Hart, Maiah H Dent, Paolo Fortina, C Patrick Reynolds, John M Maris
Scientific data  ·  28 Mar 2017  ·  pubmed:28350380

2016

International Comparison of Enumeration-Based Quantification of DNA Copy-Concentration Using Flow Cytometric Counting and Digital Polymerase Chain Reaction.
Hee-Bong Yoo, Sang-Ryoul Park, Lianhua Dong, Jing Wang, Zhiwei Sui, …, Jim F Huggett, Helen Parkes, Margaret C Kline, Jo Lynne Harenza, Peter M Vallone
Analytical chemistry  ·  30 Nov 2016  ·  pubmed:28193036
Identification of quantitative trait loci and candidate genes for an anxiolytic‐like response to ethanol inBXDrecombinant inbred strains
A. H. Putman, A. R. Wolen, J. L. Harenza, R. K. Yordanova, B. T. Webb, E. J. Chesler, M. F. Miles
Genes, Brain and Behavior  ·  01 Apr 2016  ·  doi:10.1111/gbb.12289

2015

Inhibition of monoacylglycerol lipase reduces nicotine withdrawal
P P Muldoon, J Chen, J L Harenza, R A Abdullah, L J Sim‐Selley, B F Cravatt, M F Miles, X Chen, A H Lichtman, M I Damaj
British Journal of Pharmacology  ·  08 Jan 2015  ·  doi:10.1111/bph.12948

2014

The α3β4* nicotinicAChreceptor subtype mediates physical dependence to morphine: mouse and human studies
P P Muldoon, K J Jackson, E Perez, J L Harenza, S Molas, …, M Dierssen, M F Miles, X Chen, M De Biasi, M I Damaj
British Journal of Pharmacology  ·  25 Jul 2014  ·  doi:10.1111/bph.12741

2013

Genetic variation within theChrna7gene modulates nicotine reward‐like phenotypes in mice
J. L. Harenza, P. P. Muldoon, M. De Biasi, M. I. Damaj, M. F. Miles
Genes, Brain and Behavior  ·  26 Dec 2013  ·  doi:10.1111/gbb.12113

2011

The Neuropeptide Galanin and Variants in the GalR1 Gene are Associated with Nicotine Dependence
Kia J Jackson, Xiangning Chen, Michael F Miles, JoLynne Harenza, M Imad Damaj
Neuropsychopharmacology  ·  27 Jul 2011  ·  doi:10.1038/npp.2011.123
Genetic analysis of the neurosteroid deoxycorticosterone and its relation to alcohol phenotypes: identification of QTLs and downstream gene regulation.
Patrizia Porcu, Todd K O’Buckley, Soomin C Song, Jo Lynne Harenza, Lu Lu, Xusheng Wang, Robert W Williams, Michael F Miles, A Leslie Morrow
PloS one  ·  08 Apr 2011  ·  pubmed:21494628