Vania Broccoli, PhD, is a developmental neurobiologist who has been investigating some crucial molecular mechanisms shaping brain development and neuronal differentiation. He, then, has translated some of this knowledge in the stem cell research field contributing to develop protocols and methods for generating sub-type specific telencephalic neurons through in vitro differentiation of embryonic and neural stem cells.
Lately, his group employed lineage-specific transcription factors for reprogramming cellular identity and generating therapeutic relevant neuronal subtypes from conversion of skin fibroblasts. Initially, he established genetic cell reprogramming for generating iPS cells with the aim to model human diseases like Alzheimer’s and Parkinson’s disease and atypical Rett syndrome. Then, his group identified a minimal combination of three transcription factors (Mash1, Nurr1 and Lmx1a) able to directly convert model and human fibroblasts into functional dopaminergic neurons. This discovery allows for the straightforward production of a homogenous source of human functional dopaminegic neurons amenable for a cellular replacement therapy in Parkinson’s disease and its in vitro modeling.
Expression of GCase throughout Brain Vasculature for Degradation of Alpha-synuclein Aggregates
Direct Reprogramming of Functional, Induced Dopaminergic Neurons from Human Adult Somatic Cells