March 09, 2008
Improved Gene Therapy For Embryonic Stem Cells

Yet another step toward clinically usable stem cells.

Donovan and Leslie Lock, assistant adjunct professor of biological chemistry and developmental and cell biology at UCI, previously identified proteins called growth factors that help keep cells alive. Growth factors are like switches that tell cells how to behave, for example to stay alive, divide or remain a stem cell. Without a signal to stay alive, the cells die.

The UCI scientists – Donovan, Lock and Kristi Hohenstein, a stem cell scientist in Donovan’s lab – used those growth factors in the current study to keep cells alive, then they used a technique called nucleofection to insert DNA into the cells. Nucleofection uses electrical pulses to punch tiny holes in the outer layer of a cell through which DNA can enter the cell.

With this technique, scientists can introduce into cells DNA that makes proteins that glow green under a special light. The green color allows them to track cell movement once the cells are transplanted into an animal model, making it easier for researchers to identify the cells during safety studies of potential stem cell therapies.

Scientists today primarily use chemicals to get DNA into cells, but that method inadvertently can kill the cells and is inefficient at transferring genetic information. For every one genetically altered cell generated using the chemical method, the new growth factor/nucleofection method produces between 10 and 100 successfully modified cells, UCI scientists estimate.

Gene therapy has been a great disappointment. Back in the mid 1990s gene therapy research seemed more promising. This gene therapy method is for cells that can be removed from the body. So it is useful for preparing stem cells (and probably non-embryonic stem cells) to accept DNA. But it is not a general solution for gene therapy.

This report is especially interesting because the improvement by orders of magnitude. To get from where we are to where we need to be with gene therapy and stem cell therapy we need many advances that bring orders of magnitude improvements in our ability to manipulate cells and genes

Share |      Randall Parker, 2008 March 09 12:32 AM  Biotech Stem Cells

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