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Disease Models & Mechanisms|May 31, 2012
Myotubular myopathy and the neuromuscular junction: a novel therapeutic approach from mouse modelsJames J Dowling, Romain Joubert, Sean E Low, et al.Pediatric Blood & Cancer|December 2, 2017
Long-term evidence that a pediatric oncology mentorship program for young investigators is feasible and beneficial in the cooperative group setting: A report from the Children's Oncology GroupAdam J Esbenshade, Christopher R Pierson, Amanda L Thompson, et al.Human Molecular Genetics|November 10, 2011
Modeling the human MTM1 p.R69C mutation in murine Mtm1 results in exon 4 skipping and a less severe myotubular myopathy phenotypeChristopher R Pierson, Ashley N Dulin-Smith, Ashley N Durban, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|July 23, 2023
Novel MECP2 gene therapy is effective in a multicenter study using two mouse models of Rett syndrome and is safe in non-human primatesSamantha Powers, Shibi Likhite, Kamal K Gadalla, et al.Acta Neuropathologica Communications|November 21, 2022
Molecular and spatial heterogeneity of microglia in Rasmussen encephalitisJesse J Westfall, Wesley N Schwind, Sahibjot Sran, et al.American Journal of Human Genetics|August 5, 2014
SPEG interacts with myotubularin, and its deficiency causes centronuclear myopathy with dilated cardiomyopathyPankaj B Agrawal, Christopher R Pierson, Mugdha Joshi, et al.European Journal of Medical Genetics|June 14, 2019
Expanding the clinical history associated with syndromic Klippel-Feil: A unique case of comorbidity with medulloblastomaKathleen M Schieffer, Elizabeth Varga, Katherine E Miller, et al.The American Journal of Pathology|April 15, 2014
Differential muscle hypertrophy is associated with satellite cell numbers and Akt pathway activation following activin type IIB receptor inhibition in Mtm1 p.R69C miceMichael W Lawlor, Marissa G Viola, Hui Meng, et al.Journal of Neuropathology and Experimental Neurology|October 19, 2019
GOPC-ROS1 Fusion Due to Microdeletion at 6q22 Is an Oncogenic Driver in a Subset of Pediatric Gliomas and Glioneuronal TumorsTimothy E Richardson, Karen Tang, Varshini Vasudevaraja, et al.Pediatric Blood & Cancer|July 17, 2019
Mentors' perspectives on the successes and challenges of mentoring in the COG Young Investigator mentorship program: A report from the Children's Oncology GroupAdam J Esbenshade, Lisa S Kahalley, Reto Baertschiger, et al.Pageof 11