Boston Children's Hospital's Human Neuron Core offers a new paradigm for disease modeling and preclinical drug screening in an academic setting. The mission of the core is to bridge the gap between the clinic and basic researchers and help establish human induced pluripotent stem cell (iPSC)-derived model systems for neurological disorders as well as provide support for preclinical research developing screens to identify novel therapeutics. 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.
The HNC exploits transformative stem cell technology for both modeling of specific diseases and screening of test compounds in human neurons derived from 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
6. compare results of these "pre-clinical" drug trials with clinical trials in patients.
Dosh Whye, BA MA
Assistant Director
Human Neuron Differentiation Services
Human Neuron Core
Boston Children’s Hospital
Center for Life Science Building
3 Blackfan Circle, Boston, MA 02115
Tel: (617) 919-3593
Email: dosh.whye@childrens.harvard.edu
The HNC exploits transformative stem cell technology for both modeling of specific diseases and screening of test compounds in human neurons derived from 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
6. compare results of these "pre-clinical" drug trials with clinical trials in patients.
Dosh Whye, BA MA
Assistant Director
Human Neuron Differentiation Services
Human Neuron Core
Boston Children’s Hospital
Center for Life Science Building
3 Blackfan Circle, Boston, MA 02115
Tel: (617) 919-3593
Email: dosh.whye@childrens.harvard.edu
