Single Mitochondrion Analysis and Engineering for Human Neurological Disease

2017 Scientific Innovations Award
James Eberwine, Ph.D.
Systems Pharmacology and Translational Therapeutics
University of Pennsylvania Perelman School of Medicine

The goal of Dr. Eberwine’s proposal is to enable new therapeutic modalities for treating mitochondrial diseases through the development and use of novel mitochondrial engineering procedures. While mammalian genome engineering has been performed for decades, advances in mitochondria engineering have been hindered because little is known about individual mitochondrial differences and it hasn’t been possible to easily insert or delete genes into the mitochondria to assess potential biological roles of these differences. Even if modification were possible, there are hundreds of mitochondria in a cell, making it difficult to envision how to modify all mitochondria in the same way. This application details novel approaches to overcome these issues, providing a technical and theoretical framework to create therapeutic mitochondria and enhance the understanding of basic mitochondrial function. In particular Dr. Eberwine’s lab will detect, quantify and functionally assess specific single mitochondrion mutations in mouse and human neurons and astrocytes, in hopes that these data will provide new insights into the increasingly complex role mitochondria play in modulating neuronal cell functioning.

Other Awards

James J DiCarlo, M.D., Ph.D., Massachusetts Institute of Technology
Using Computer Models of the Neural Mechanisms of Visual Processing to Non-Invasively Modulate Brain States
DiCarlo’s research team is exploring an innovative approach to address emotional challenges, such as anxiety and depression. Traditional treatments for these disorders can be complex and often cause unpleasant side effects,…
Eiman Azim, Ph.D., The Salk Institute for Biological Studies
Learning from Error: Defining how Cerebellar Circuits Drive Adaptation in a Changing World
The ability to move effectively through the world is one of the most important functions of the brain. However, the world and the body are constantly changing, meaning the signals…
Hillel Adesnik, Ph.D., University of California, Berkeley
All Optically Probing the Neural Codes of Perception in the Primate Brain
How patterns of action potentials in space and time give rise to sensory experience is among the most enduring mysteries of biology. Despite decades of experiments correlating brain activity patterns…
Chaolin Zhang, Ph.D., Columbia University
Human-specific Alternative Splicing, Brain
Development, and Ciliopathies
Like movie frames needing to be edited to tell an engaging story, pieces of genetic information stored in DNA for each gene need to be sliced and rejoined, through a…