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Nanotechnology Research Lab: Nanomedicine Center for Nucleoprotein Machines

Some of the nanotechnology rsearch in medicine focuses on treating symptoms of diseases, not the underlying genetic causes. The Nanomedicine Center for Nucleoprotein Machines, started in 2006, is one organization that is focused entirely on the genetic causes of disease. Their hope is that one day many common diseases will be treated directly at the genetic level.

The Center is one of six nanomedicine development centers funded by the U.S. National Institutes of Health. Participating institutions are Georgia Institute of Technology, Stanford University, New York University, Cold Spring Harbor Laboratory, Emory University, Harvard Medical School, Medical College of Georgia, and MIT.

This Center is interesting in that it is focused on one challenging issue, which they state as “understanding and re-directing natural processes for repair of damaged DNA.” The Center’s five-year goal is to reengineer the ”homologous recombination repair machine” to provide a clinically applicable gene correction technology.”

The Nucleoprotein Machines part of the Center’s name relates to the fact that proteins are nature’s workhorses for repairing damaged DNA. A nucleoprotein is simply a protein that is attached to a nucleic acid such as DNA.

Homologous recombination is a form of genetic repair. When a break occurs in DNA, double-strand homologous recombination is a natural process in which the structure of a neighboring DNA is copied onto the damaged DNA to repair it. This process occurs constantly in our bodies to repair naturally occurring damage to DNA.

The current focus at the Center involves producing a gene correction device in the form of an engineered protein. This protein is designed to detect a defective DNA double strand and cut off the defective portion. After the DNA double strand has been cut, it can be repaired by the natural homologous recombination process.

The Center is testing its methods on sickle-cell disease in mice. Sickle-cell is a genetic disease that causes red blood cells to have an abnormal shape. This odd shape results in the cells moving through the bloodstream less easily than normally shaped red blood cells do, which results in anemia.

Sickle-cell, which has no known cure, is a painful disease that shortens life. The Center uses mice and models of sickle-cell disease to find a way to repair the mutated gene that causes it. This focus on sickle-cell could lead to repair of other single-gene diseases.

According to the Center’s 2010 Progress Report — Executive Summary, the areas of development are as follows:

  • Development of nanoprobes to investigate the assembly, disassembly, and control of DNA repair machines. Machines in genes manage the storage of information about cells. The Center has a goal of learning how to modify the information stored in DNA and RNA. Initial work has begun, though they acknowledge that solving the puzzle could take decades.

  • Development of new tools to create a model for nanomachines to assist in the repair of DNA double-strand breaks. Researchers will use these machines to understand the functions of DNA and eventually learn how to manipulate it.

  • Development of strategies for tagging components of DNA and RNA.

  • Synthesis of quantum dots (semiconductor nanocrystals) that are less bulky than those commercially available.

  • Development of small beacons that offer the means to image protein interactions. The Center is the first to capture real-time images of certain proteins.

  • Development of a method of delivering proteins to the nucleus of human cells.

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