Researchers are now finding new ways to deliver gene therapy without using viruses as carriers. Researchers are using 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. 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.
AdvertisementWhen the lipid/DNA globule is taken up by a cell, it's moved to an acid-filled compartment called the endosome. A key step here, 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. Thus researchers conclude saying "What we're doing is reinventing a virus through chemistry ".