The Human Neuron Differentiation Service (HNDS) is one component of the Human Neuron Core, comprised of the HNDS and the Assay Development and Screening Facility (ADSF), that 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.
We provide reproducible quality-controlled preparations of neurons that release preclinical research lab personnel from labor required for neuron production to focus on the execution of cutting edge research. The core can receive iPSC lines under IRB approval from patients characterized by clinicians in other Massachusetts hospitals. The phenotyping service will be of particular value to clinical investigators who work closely with patients, but may lack basic science skill sets needed to establish iPSC lines, derive specific neurons from these and conduct sophisticated phenotypic analysis.