Researchers at UCI Study Genetic Alteration to Prevent Disease

The UC Irvine Sue and Bill Gross Stem Cell Research Center is researching genetic alteration in order to potentially cure hereditary diseases. While researchers are primarily focused on examining genomes (full sets of chromosomes) in order to diagnose fatal genetic diseases, technology that analyzes DNA strands may permit scientists to examine individual characteristics of each embryonic cell, beckoning the question of whether or not these traits can be changed.
The concept of changing hereditary characteristics in babies was recently addressed in a CNN article dated Oct. 30. “Designer babies,” said CNN reporter Mike Steere, “could be well possible in the near future.”
While researchers at the Sue and Bill Gross Research Center agree that this technology is becoming more and more possible, they doubt whether it will actually be used.
According to Aileen Anderson, Ph.D and associate professor in the Physical Medicine and Rehabilitation department, while the technology to look at the genomes and search for specific disease is there, the ability to actually go in and change human DNA is not.
“You’ll know a lot about what the potential for an individual might be [even before birth],” Anderson said. “But this is not going to happen in the next 10-20 years. It is more likely to happen over the next 100 years which will give people time to grapple with it; and I think our sense of scope is going to change over time. I suspect it’s not going to be what it sounds like in the movies; I suspect that people are going to shy away from that kind of elimination.”
While creating designer humans is not on the list of priorities, stem cell researchers have experimented with changing animal DNA by producing fluorescent green rats and mice. However, Brian Cummings, associate professor in the Physical Medicine and Rehabilitation department, said they would never treat humans in the same way.
Furthermore, the research being done at the center is not related to “designing babies.” Rather, it studies genetic alteration in order to study specific genetically associated diseases.
According to Vice Chancellor for Research Susan Bryant, UCI’s stem cell researchers are looking at stem cells for signs of Alzheimer’s disease, diabetes, Huntington’s disease and even spinal cord injury.
While Alzheimer’s disease and diabetes are currently difficult to treat or predict through DNA, stem cell research will soon have a profound effect on strictly hereditary diseases that are immediately present at birth.
Batten disease, for example, is a life-limiting hereditary disease in which a child lives and grows normally until a certain age, usually around age 4, when they will suddenly begin to deteriorate and digress due to required enzymes being absent. While Batten is a tragic disease, it is an excellent candidate for in utero treatment, which involves treatment using stem cells while the baby is still in the uterus.
“In the case of storage diseases like this,” Anderson said, “in utero treatment is the earlier the better. You would treat in utero with the stem cell derived from that child and engineered with the spinal infector to express the enzymes that have been missing so that you’re incorporating during development the needed enzymes that have been missing. Over the next 10-20 years, this will mean enormous benefits for small populations of people.”
Developments are mainly done with pluripotent stem cells, cells with the potential to become any type of cell in the body. These cells, Assistant Professor of Pathology Edwin Monuki said, have the lofty goal of changing into any kind of cell in order to replace organs or tissues in the body that are either tired and worn out or diseased.
In several different labs, scientists are testing the ability of changing normal non-stem cells into stem cells by introducing only a few genes to them, forcing them, in a sense, to go back in time.
“This provides the potential of getting around the ethical problems of stem cell research because you don’t have to necessarily start with an embryo or something with the potential to become an embryo. You can actually start with an adult cell and you can add the genes to it and get it to revert to a stem cell,” Monuki said.
While these pluripotent cells may help cure disease in the future, the technology is not quite up to par. Although researchers continue to work on diseases like Alzheimer’s and diabetes, they find that they are more difficult to predict in utero because the diseases themselves do not present themselves until years after the child is born.
Scientists, instead, choose to look at stem cells that have the same genetic problems that they expect with Alzheimer’s or Lou Gehrig’s, another disease that damages the nerves, and use the cells in the culture and subculture systems to experiment with drugs in order to alter their behavior.
“Whether you’re going to treat the disease using stem cells, is not the same question as whether you’re going to study the disease using stem cells,” Cummings said. “You can find a way to treat the fetus before it’s born and transplant cells into the womb. So that may be one way that stem cells might be used to treat diseases.”
Cummings added that the research center does not want to look at diseases that are not immediately fatal for fear of destroying parts of the genomes without knowing enough about them.
“If somebody has heart disease, which is certainly genetically linked, we still don’t know enough about the genome yet to say the things that make someone predisposed to make someone likely to get heart disease when they’re 50-years-old,” Cummings said. “Maybe that gene is also related to how smart they are — who the hell knows? Are you going to take that gene out and replace it in utero so that person doesn’t get heart disease, but could end up being retarded and whatnot? That’s not where we want to go; we want to study diseases that are fatal.”