A5035214295- Nuclear Receptors and Signaling
- RNA regulation and disease
- Pluripotent Stem Cells Research
- Neuroscience and Neural Engineering
- RNA Interference and Gene Delivery
- Macrophage Migration Inhibitory Factor
- Nerve injury and regeneration
- Autism Spectrum Disorder Research
McLean Hospital
2019-2023
Harvard University
2019-2023
Massachusetts General Hospital
2019
Harvard Stem Cell Institute
2019
Parkinson's disease (PD) is a neurodegenerative disorder associated with loss of striatal dopamine, secondary to degeneration midbrain dopamine (mDA) neurons in the substantia nigra, rendering cell transplantation promising therapeutic strategy. To establish human induced pluripotent stem cell–based (hiPSC-based) autologous therapy, we report platform core techniques for production mDA progenitors as safe and effective product. First, by combining metabolism-regulating microRNAs...
The nuclear receptor, Nurr1, is critical for both the development and maintenance of midbrain dopamine neurons, representing a promising molecular target Parkinson's disease (PD). We previously identified three Nurr1 agonists (amodiaquine, chloroquine glafenine) that share an identical chemical scaffold, 4-amino-7-chloroquinoline (4A7C), suggesting structure-activity relationship. Herein we report systematic medicinal chemistry search in which over 570 4A7C-derivatives were generated...
Parkinson's disease (PD) is characterized by the selective death of substantia nigra pars compacta (SNpc) dopaminergic neurons and includes both motor non-motor symptoms. While numerous models exist for study typical PD deficits, fewer Previous studies have shown that a Pitx3-/- mouse model (aphakia or ak mouse) has specific developmental failure neuron population in SNpc it can be used PD-related gross dysfunction as well cognitive functional deficits. It remains unclear whether aphakia...
Abstract BACKGROUND Surgical implantation of cellular grafts into the brain is increasing importance, as stem cell-based therapies for Parkinson and other diseases continue to develop. The effect grafting technique on development survival graft has received less attention. Rate method delivery may impact cell viability success these therapies. Understanding final location with respect intended target also critical. OBJECTIVE To describe a “columnar injection” designed reduce damage host...