Science&Health: Genetic researchers seek to cure diseases

Scientists working on genetics research may be on their way to understanding the foundations of being genetically free from disease, identifying favored traits, and allowing doctors to treat previously incurable illnesses.

UCLA researchers, along with others around the country, are currently using the vast amounts of genetic information now available to learn more about the body’s mechanisms for growth and maturation. They are looking to pinpoint sites to prevent the development of certain diseases.

Geneticists are able to study DNA to isolate sequences called genes, which code for protein, that either help or hinder the body’s defence against disease. Once these gene sequences are better understood, scientists will be able to better detect and identify causes of diseases such as cancer, said Stanley Nelson, professor of pediatrics and human genetics.

“We look at the genetic mutations that occur in cells that can lead to cancer, and can now develop technologies that will determine which genes are key factors. If you know which genes, the cancer can be subverted (and) we can target where to try to inhibit those tenancies,” Nelson said.

Looking specifically at the genetic sequences allows scientists to find the sequences that cause disease and insert genetic information to interrupt those diseases. For example, scientists can interrupt SCID, a severe immune system deficiency, “so children who would have had to live in a bubble all their lives no longer have to,” said James Walter, O’Malley professor of bioethics and chair of the Bioethics Institute at Loyola Marymount University.

Better understanding the causes of specific diseases furthers the research to study genetic causes of cancer more efficiently, Nelson said.

“With all the genetic information available, we can systematically look to see what genes cause different cancers,” Nelson said. “If we can find which genes are most important for different types of cancers, that may differ very dramatically at the molecular level and enable us to diagnose the cancers more clearly.”

Scientists use mice as models for humans, testing how genetic alterations can hinder the progression of certain diseases.

Studies are also being conducted at the cellular level to understand the molecular signaling pathways that determine how an undifferentiated cell will mature and specialize in the body and how scientists may be able to enhance healthy traits.

“With the issue about genetic manipulation to cure disease or to enhance an individual, there are a number of factors to consider: What is a “˜better’ genetic trait? We can’t decide as a society who even counts as a person; who is going to decide what “˜better’ is?” Walter said.

While the ability to target genetic sequences in order to introduce or enhance traits is not yet possible, bioethics researchers suggest a discussion is needed before the technology becomes available.

“There are a lot of inequalities built into (selectively enhancing traits). I am not saying that it is the wrong thing to do, but there needs to be a discussion about incrementally incorporating these enhancements so that they are equally distributed,” Walter said, referring to the methodology in making these medical technologies available to all.

While decoding genes in order to enhance traits may be far in the future, UCLA scientists are currently studying the mechanisms for developing specific cell growth, said Jerome Zack, assistant director of the UCLA Institute for Stem Cell Biology and Medicine.

The institute works “to collaborate and coordinate researchers studying stem cells, and it is designed to bring scientists together so that they reach goals faster than if they worked on their own,” Zack said.

These scientists have found that stem cells differ from other body cells most significantly when they divide. Rather than splitting into two identical cells, one of the new cells matures and differentiates while the other remains a stem cell. The remaining stem cell is able to replenish the body’s source of stem cells, unlike other cells in the body, Zack said.

Signal pathways mark the stem cell’s course to growing into an adult differentiated cell.

Scientists from the institute are now studying the signal pathways inside the cell that determine how the cells specialize. They hope to use those mechanisms to cure defects or halt disease.

In the future, scientists hope to elucidate the signal pathway to develop whole organs, so that in theory, scientists could create a whole new heart and use it to replace a diseased heart, thereby correcting heart disease.

“Certainly, people are interested in (whether) you can create nervous system cells, brain tissues or nerve tissues to repair spinal cord injuries or cure Alzheimer’s disease,” Zack said.

“This is, right now, a dream. We are not there yet, but if we learn the signals, we can get there,” he added.