Putting Genes In A Pill

[Murray Charters <mcharters@xxxxxxxxxxxxxx>]

Putting Genes In A Pill
Source: University Of California - Davis
Date: 2003-05-15

Finding new ways to deliver gene therapy without using viruses as
carriers is the aim of research by chemist Michael Nantz at UC Davis.

Nantz's lab engineers lipids, oily molecules that can form a
protective complex around DNA, to do the same job as a virus. The
lipids protect the DNA and help it get into the target cell. The
approach could eventually make gene therapy treatments that are
stable enough to take as a pill.

Gene therapy holds the promise of curing diseases such as diabetes,
cystic fibrosis or Parkinson's disease by replacing damaged or
missing genes. To do that, a new piece of DNA has to be carried into
cells in the right part of the body.

Most approaches have used some kind of genetically modified virus to
carry the DNA into the cell. But safety issues have been raised about
that approach following the 1999 death of a patient at the University
of Pennsylvania and cases of leukemia in two French children being
treated for "Bubble baby syndrome."

When the lipid/DNA globule is taken up by a cell, it's moved to an
acid-filled compartment called the endosome. A key step, Nantz said,
is to engineer lipids that can get the DNA out of the endosome and
into the cell nucleus where most of the genetic material resides.

"What we're doing is reinventing a virus through chemistry," he said.


In 1998, Nantz helped found a biotech company, Genteric Inc., to
develop oral delivery systems for gene therapy. Because cells in the
gut turn over quickly, the effect of the gene therapy drops over
time. So the treatments can act more like a conventional medicine
than a permanent genetic fix.

Editor's Note: The original news release can be found here.


Note: This story has been adapted from a news release issued for
journalists and other members of the public. If you wish to quote any
part of this story, please credit University Of California - Davis as
the original source. You may also wish to include the following link
in any citation:

SOURCE: Science Daily


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