Skin Cells Converted Into Neurons That Affect Huntington’s Disease

MedicalResearch.com Interview with:
Matheus Victorm PhD candidate
Dr. Andrew Yoo’s Lab
Graduate Program in Neuroscience
Washington University School of Medicine

Medical Research: What is the background for this study? What are the main findings?

Answer: We have described a way to convert human skin cells directly into a specific type of brain cell called medium spiny neurons that is affected by Huntington’s disease, an ultimately fatal neurodegenerative disorder. Unlike other techniques that turn one cell type into another, this new process does not pass through a stem cell phase. Furthermore, we demonstrated that these converted cells survived at least six months after injection into the brains of mice and behaved similarly to native cells in the brain. Not only did these transplanted cells survive in the mouse brain, they showed functional properties similar to those of native cells. These cells are known to extend projections into certain brain regions and we found the human transplanted cells also connected to these distant targets in the mouse brain.

Medical Research: What should clinicians and patients take away from your report? What recommendations do you have for future research as a result of this study?

Answer:  The generation of a highly enriched population of medium spiny neurons allows the possibility of dissecting the pathogenesis of Huntington’s disease in a dish. This will enable researchers to investigate potential new drugs or small molecules that could be used to treat patients suffering from this disorder.To study the cellular properties associated with the disease, we are now taking skin cells from patients with Huntington’s disease and reprogramming them into medium spiny neurons using the approach described in the new paper. We also plan to inject healthy  reprogrammed human cells into mice with a model of Huntington’s disease to see if this has any effect on the symptoms.

Citation:

Generation of Human Striatal Neurons by MicroRNA-Dependent Direct Conversion of Fibroblasts

Matheus B. Victor,Michelle Richner, Tracey O. Hermanstyne, Joseph L. Ransdell, Courtney Sobieski, Pan-Yue Deng, Vitaly A. Klyachko, Jeanne M. Nerbonne, Andrew S. Yoo

Neuron DOI: http://dx.doi.org/10.1016/j.neuron.2014.10.016

 

 

Last Updated on October 24, 2014 by Marie Benz MD FAAD