Persistent Gene Transfer to Mitochondria
Abstract
The United Mitochondrial Disease Foundation (UMDF) estimates that ?every 30 minutes, a child is born who will develop a mitochondrial disease by the age of 10.? This corresponds to a population of 1,000 to 4,000 affected children born each year in the United States. Mitochondrial diseases can affect overall energy supply as well as specifically targeted metabolic pathways. For these reasons, it is critical that interventions have the ability to ?grow? with patients and, if possible, compete effectively with defective mitochondrial genomes for control of mitochondria. Among the many types of problems that mitochondria can develop, perhaps the most inaccessible problems are caused by mutations that develop in the subset of human DNA that is hidden in mitochondria instead of the cell?s nucleus. Recent advances have provided a proof-of-principle that genes can be delivered to mitochondria. Given the availability of this technology, it is critical that we address the issue of developing therapies that persist in patients. The present proposal describes a plan to investigate whether virus-delivered genes can be taught to grow along with the mitochondria that they enter. Should this project be successful, the resulting knowledge could be applied to many genetic therapies that will be targeted at specific defects in the mitochondrial genome.
Document Details
- Document Type
- DoD Grant Award
- Publication Date
- Aug 07, 2017
- Source ID
- W81XWH1710379
Entities
People
- Philip Leopold
Organizations
- Stevens Institute of Technology
- United States Army