Rosamund Stone Zander Translational Neuroscience Center (RSZ TNC) | Cores

Our resources

The unique environment at Boston Children’s Hospital provides unprecedented opportunities for the development of new diagnostic tools and therapies. The center’s current cores and facilities include a Biorepository Core, Human Neuron Differentiation Service, Clinical Research and Regulatory Affairs Service, Human Neurobehavioral Core Service, Cellular Assay Development and Screening Core and Mouse Neurodevelopmental Behavior Core, with many more cores in development.

RSZ TNC members at Boston Children’s and their collaborators have access to a broad range of resources and expertise throughout the hospital, including:

  • diverse populations of well-characterized patients with brain disorders
  • genetics and genomics capabilities
  • MRI and radiology expertise
  • bioinformatics
  • tissue banks
  • animal models
  • stem cell platforms
  • electrophysiology
  • cognitive neuroscience laboratories
  • deep knowledge of rare diseases, both clinical and scientific
  • top clinical and basic scientists

Human Neuron Core

Mustafa Sahin, MD, PhD, Director  

The Human Neuron Core at Boston Children’s Hospital offers a new paradigm for disease modeling and preclinical drug screening informed by broad expertise at Harvard in neuroscience, neurology and stem cell biology. Generous funding from the Massachusetts Life Sciences Center has enabled us to significantly expand our equipment and services and open them to the broader research community.

This core will exploit transformative stem cell technology for both modeling of specific diseases and screening of test compounds in human neurons derived from induced pluripotent stem cells (iPSCs). The goals of this service are to: (1) develop standard operating procedures for the generation of different types of neuronal cell lines; (2) create neuronal cell lines from iPSCs derived from patients with specific diseases and from healthy controls; (3) compare key characteristics (e.g. shape, growth, synaptic connectivity, protein composition) of patient-derived and control-derived neurons; (4) identify disease-specific characteristics in patient-derived neurons; (5) screen drug candidates in disease-specific cell lines to greatly increase the speed and specificity of drug discovery; and (6) compare results of these “pre-clinical” drug trials with clinical trials in patients. 

Services fall in three main categories 

  • The Human Neuron Differentiation Service provides well-characterized human neurons to academic investigators and industry collaborators to support translational research programs. Cell types available include human neurons derived from induced pluripotent stem cell (iPSC) lines, including excitatory cortical neurons, GABA neurons, motor neurons, dopaminergic neurons and sensory neurons.
  • The Human Neuron Phenotyping Service can compare key cellular characteristics across patient-derived and control-derived neurons. Phenotyping services include use of high content measures of neuronal morphology, neuronal excitability using multiple electrode array recordings and targeted RNA sequencing profiles (AmpliSeq) of neuronal transcriptomes.
  • The expanded Assay Development Screening Facility provides multiple live-cell screening platforms that can assess image- and activity-based measures of live neurons hourly, in a medium/high-throughput capacity. Key equipment available for hourly use includes the Hamamatsu Functional Drug Screening System, the ArrayScan XTI: High content screening with live cell imaging and a range of robotics and liquid handlers to support drug screening.

Preclinical Consulting

Meera Modi, PhD, Assistant Director

Dr. Modi has extensive drug discovery experience in both industry and academia. She is available to consult with BCH investigators on developing strategies and finding resources to translate their basic science findings into therapeutic approaches to treat pediatric brain disorders.

Human Neurobehavioral Core Service

Charles A. Nelson, PhD, and Deborah Waber, PhD, Co-Directors

The goal of the Neurobehavioral Core Service is to improve access to and expand behavioral assessment resources for translational neuroscience research at Boston Children’s Hospital. The core represents a collaboration between the Laboratories of Cognitive Neuroscience and Neurodevelopmental Disorders Phenotyping Program in the Division of Developmental Medicine and the Clinical Behavioral Science Core in the Department of Psychiatry. This RSZ TNC core aims to offer a standardized set of state-of-the-art assessments of children — from infancy to young adults. 

The core directors will also provide guidance for researchers on:

  • which standardized tests are likely to provide the best information for a particular clinical research study
  • which specialized laboratories at Boston Children’s might provide novel tests 
  • which specialized tests are available at other local institutions in Boston
  • how to approach the development of a new test
  • which tests might provide the best translation from animal studies to human studies 

Repository Core for Neurological Disorders (Click Here For More Information)
Mustafa Sahin, MD, PhD, Lindsay Swanson, MS, and Kira A. Dies, ScM, CGC, Co-Directors

The Repository Core for Neurological Disorders is a Boston Children’s Hospital database and biological specimen bank that pairs biological samples with phenotypic data for translational research and clinical trials. To better understand causes and create new therapies for neurological disorders, it is often necessary for researchers to investigate various patients with the same or similar conditions. Therefore, such studies can benefit immensely from access to a database of high-quality clinical data and paired DNA and protein samples from affected individuals. This database and bio-specimen bank can save both time and resources that would be required to collect these data independently. As a result, all data, DNA and other bio-samples will be stored indefinitely and will potentially be used by multiple investigators for future studies of the causes and/or treatment of neurological disorders. The Core is anticipated to lead to new and enhanced collaborations among users, including collaborations between those who submit and those who access the data, between those who analyze the data with different primary goals, and between those using this and other Cores. Any patient with a neurological disorder and their family members are eligible to participate.

Clinical Research and Regulatory Affairs Service
Kira A. Dies, ScM, CGC, and Stephanie Jo Brewster, MS, CGC, Co-Directors

This service facilitates the mission of the Rosamund Stone Zander Translational Neuroscience Center, providing coordination among studies, communications, resource development and implementation of new or ongoing clinical studies. Additionally, the co-directors serve as leadership for the Neuroscience Clinical Research Cluster and the Translational Core of the Intellectual and Developmental Disabilities Research Center (IDDRC) for Boston Children’s Hospital. The service is led by experts in protocol development and launching of new studies.  Staff are available  to guide RSZ TNC researchers in designing human studies, including the preparation of Institutional Review Board (IRB) and FDA submissions. Additionally, staff of the Clinical Research and Regulatory Affairs Service help researchers with recruitment plans, budget development, supervision of study coordinators, study monitoring and audit preparation.

Our services include:

We offer a variety of services to clinical research teams within the hospital. These include:

  • regulatory guidance around federal, state and institutional policies including Good Clinical Practice
  • consultation regarding protocol development
  • facilitation of Institutional Review Board (IRB) and FDA submissions 
  • implementation of new studies 
  • project management
  • development of training materials and workshops for study personnel
  • facilitation of patient recruitment

Through monthly program managers’ meetings, we regularly evaluate best practices in the conduct of clinical research. We also facilitate access to other RSZ TNC resources including the Human Neurobehavioral Core Service and Biorepository, as well as additional research resources at Boston Children’s.  

 Our track record:

Recent projects we have facilitated include:

Industry-sponsored clinical trials

  • Phase II trial of MG01CI treatment in Fragile X syndrome 
  • Phase II trial of RO5186582 treatment in Down syndrome 
Investigator-initiated clinical trials

  • Phase IIb trial of RAD001 treatment in Tuberous Sclerosis Complex 
  • Phase II trial of human IGF-1 treatment in Rett Syndrome 
Multi-site studies

  • Biomarkers in Autism Spectrum Disorder 
  • Phase II trial of N-Carbamylglutamate treatment in Acute Hyperammonemia 
  • Everolimus treatment in PTEN clinical trial

For more information, contact Kira Dies at or Stephanie Brewster at

Neurophysiology Services
Susan Faja, PhD, Alexander Rotenberg, MD and Jurriaan Peters, MD, Co-Directors

The Neurophysiology Service aims to become a leader in the facilitation of acquisition and processing of high quality human neurophysiological data reflecting underlying pathophysiology, and of potential use as surrogate biomarker, in neurologic disease and developmental disorders. Through routine and timely delivery of the highest quality neurophysiological data, including EEG data, and strict adherence to ethical research standards, we aim to accelerate translational research and clinical trials and allow for next generation interventions for neurological disorders. The Core provides consultation regarding trial design, rationale for a neurophysiology measure relevant to target disorder or developmental process, assessment of data-acquisition needs including contextual review of the literature and current competitive efforts elsewhere, identification of research collaborators, and recommended software or platform for processing.

 We offer a variety of services to clinical research teams within the hospital. These include:
 We offer a variety of services to clinical research teams within the hospital. These include: