St. Jude Research
CPNDR

Center for Pediatric Neurological Disease Research

Director: Peter J. McKinnon, PhD

About CPNDR

The Center for Pediatric Neurological Disease Research (CPNDR) is tasked with advancing basic research investigating complex, pressing questions relating to pediatric neurological diseases. CPNDR stands as a fundamental program of the PTNI, upstream to the efforts of the Center for Experimental Neurotherapeutics (CENT). CPNDR is the source of discovery –  identifying what genes are involved in disease pathogenesis, what function those genes serve, how mutation in those genes drives disease, and whether we can model these mutations for preclinical evaluation. CPNDR serves as an anchor program with a critical mass of investigator-driven research programs sharing physical proximity and access to specialized resources. CPNDR and CENT engage in bidirectional translational efforts, shepherding molecular clues to therapeutic investigation, and sourcing clinical observations to inform basic research. 

CPNDR faculty take their research cues from pediatric neurological diseases, directing programs intended to discover, define, and alter these disorders. 

Research Faculty

Research
programs

  1. Genome stability in pediatric neurodegeneration

    Genome instability is directly connected to a myriad of diseases, either as a primary cause or a cofounding secondary event, and in many neurodevelopmental disorders and later onset neurodegenerative events. We strive to understand the disease etiology of DNA damage and genome maintenance pathways, and the relationship to chromatin structure and regulation. We are also interested in deciphering cell type specificity and genome stability as it relates to neurological disease pathogenesis. 

    Visit the McKinnon Lab

  2. Genetics of neurodevelopmental disease and epilepsy in children

    Seizures that begin in the first weeks to years of a child’s life can be difficult to treat and are associated with poor developmental outcomes, developmental delays, intellectual disability, and autism spectrum disorder. We are focused on investigating the genetic basis of rare forms of epilepsy and associated neurodevelopmental disorders. Our goal is to identify novel mutations and determine their molecular and functional implications to help inform precision therapeutic development.

    Visit the Mefford Lab

  3. Astrocyte regulation of synaptic balance in brain development

    Proper brain development relies on the precise formation and function of neuron-neuron connections, known as synapses, and correct brain function relies on a tightly regulated balance between excitatory and inhibitory synapses. We investigate the cellular and molecular mechanisms of astrocyte-neuron communication that govern this synaptic balance during brain development. By uncovering how astrocytes regulate synaptic diversity and connectivity, we aim to advance understanding of both normal brain function and the origins of neurodevelopmental disorders.

    Visit the Irala Lab

  4. RNA and cytoskeletal regulation in brain development and neurological diseases

    Neural development is a highly orchestrated process that begins in the embryo and continues into early postnatal life, guiding the formation of complex brain circuits. We investigate the molecular and cellular mechanisms that regulate neural differentiation, growth, and maturation, with a particular focus on RNA metabolism and cytoskeletal dynamics. By uncovering how these processes shape neuronal structure and function, we aim to identify molecular drivers of neurodevelopmental and neurodegenerative diseases.

    Visit the Alsina Lab

Resources and technologies

CPNDR’s 3 new resources and technologies support fundamental research into pediatric neurological disease. Investigators affiliated with CPNDR have full access to these core centers.

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