Last updated on March 17, 2014 at 9:10 EDT

First Large-Scale Computer Simulation Of Gene Therapy

April 30, 2009


“There are several barriers to gene delivery,” says Nikolaos Voulgarakis of Berkeley, the lead author on the paper. “The genetic material must be protected during transit to a cell, it must pass into a cell, it must survive the cell’s defense mechanisms, and it must enter into the cell’s guarded nucleus.”

If all of these barriers can be overcome, gene therapy would be a valuable technique with profound clinical implications. It has the potential to correct a number of human diseases that result from specific genes in a person’s DNA makeup not functioning properly — or at all. Gene therapy would provide a mechanism to replace these specific genes, swapping out the bad for the good. If doctors could safely do this, they could treat or even cure diseases like cystic fibrosis, certain types of cancer, sickle cell anemia, and a number of rare genetic disorders.


Since then, many alternatives to viruses have emerged for use in gene therapy, including synthetic molecules like “dendrimers,” a word that derives from the Greek word for “tree.” Similar to trees, dendrimers are branching molecules that are slightly positively charged. This allows them to be loaded with DNA (which is slightly negative charged) for insertion into a cell.

Dendrimers seem to offer many advantages over viruses. They may be much less toxic, and they may offer other advantages in terms of cost, ease of production, and the ability to transport very long genes. If they can be designed to efficiently — and safely — shuttle genes into human cells, then they may be a more practical solution to gene therapy than viruses.


What they uncovered were the key factors that determine the success of dendrimers as gene delivery vehicles — things like the charges of the dendrimers and their target cell membranes, the length of DNA, and the concentration of surrounding salt. Their work has illuminated some of the molecular-level details that should help clinicians design the most appropriate gene vectors.




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American Institute of Physics