Editor’s note: In the second of WRAL TechWire’s “Human Future” series by Allan Maurer, we take an inside look at ever evolving world of genomic research through the view of a top biomedical researcher at Duke University.

DURHAM – We learned a lot from the first genetics revolution, the sequencing of the entire human genome from 1997-2003. But what’s exciting now in the field is the new technology that allows changing the genome in very precise ways, said Charlie Gersbach, PhD, a department of biomedical engineering professor at Duke University.

The powerful gene editing tool CRISPR-Cas9, which is responsible for the current revolution in genomics, only exists because of basic science research, Gersbach said during an appearance at Moogfest in Durham.

Two Nobel Prize-winning discoveries in basic science led to the discovery of the CRISPR Cas9 system, which makes it possible to target specific genetic code and edit DNA at precise locations. The CRISPR system will fuel what Gersbach called “The next genomic revolution,” helping cure nasty genetic diseases, and make science fiction-like biotechnology possible.

[VIDEO: Watch an American Scientist report about Dr. Gersbach at https://www.youtube.com/watch?v=A6W6mGiatKs ]

At a time when basic science and its funding is being questioned, it’s important to show how it results in meaningful advances, Gersbach said. Two discoveries that played a major role in developing the CRISPER system, for instance, sounded like the kinds of things politicians sometimes ridicule at first.

Why Jellyfish Glow

In one such experiment, scientists studied why jellyfish glow. “No obvious technology was seen in that,” Gersbach said. But the researchers discovered the green fluorescent protein (GFP). Inserting the gene for the protein in other animals demonstrated that a gene can be expressed throughout an organism, in given organs or in specific cells.

“That discovery now helps researchers track cells in areas such as cancer research, among many other advances in medicine, biology and technology that came from this,” Gersbach said

In the research that led directly to the CRISPR system, scientists were examining why a type of bacteria could turn off specific genes as part of a single-celled immune response. Much of the research was led by dairy industry giant Danisco, which wanted to explore how the bacteria used to make cheese and yogurt, responds to a viral attack.

“They used the GFP protein to track how the genes were getting turned off, which led to the technology (interference RNA, or RNAi) that can repurposed in humans to turn off any gene,” explained Gersbach.

The Cas9 enzyme, they found, acts like scissors that cuts DNA at a precise location.

Rare but nasty diseases

Now, using the CRISPR Cas9 tool, scientists at Duke and in thousands of laboratories globally are seeking ways to treat genetic diseases, many of which are “really horrible, nasty diseases,” Gersbach notes. They include hemophilia, the so-called Bubble Boy disease (children born with no immune system), Sickle Cell Disease, and Muscular Dystrophy.

While most are rare individually, affecting fewer than 200,000 people each, there are more than 7,000 of them collectively, affecting one in ten Americans. “There is a clear, dire, clinical need to treat these patients,” said Gersbach.

We know what the problem is in 80 percent of the cases, Gersbach said, but even with advanced gene editing and engineering tools, it is not as simple as just replacing or turning off the gene mutation causing the disease.

It’s not as if no one is making an effort. “There are 100 clinical trials every on human gene therapy,” Gersbach said. There have been more than 2,000 trials worldwide. How many led to new treatments? Not one has yet been approved despite all this effort, he added.

Safety, ethical and social questions

While the CRISPR Cas9 tool itself is so easy to make the Duke labs use high school volunteers to do it, “Getting it to do anything in any cell in a mammal is very, very difficult. Companies developing gene therapies are spending billions trying to get this to work. It is difficult to get them into human cells and get them to do what you want in human cells.”

And there are safety, ethical, and social questions involved with genetic manipulation.

“We spend a lot of time asking, ‘Is this safe?’ How do we make sure we don’t modify other cells? There are also other considerations about taking a bacterial immune system and putting it into people.”

Another question, is who is going to pay for it? “Who gets access and how will it be paid for? Two genetic treatments approved in Europe would cost $1 million in one case and half a million in another for a single treatment. On the other hand, Gersbach points out, it only takes one successful treatment to cure a genetic disease.

Delivery always a problem

“They are complicated, expensive and time-consuming to make,” Gersbach said, “so how do you charge for a drug you take one time, the patient is cured and you never see him again?”

Delivery of the treatment is always the problem, Gersbach explains. “Safety and efficiency are both connected to delivery.” Currently, most genetic treatments are delivered via a viral vector. Human cells evolved ways to keep stuff out that doesn’t belong, and viruses evolved ways to enter anyway. So the virus, stripped of harmful effects, gets the genetic treatment into the cell.

It certainly raises the question of whether we will only be able to cure genetic diseases for the well-off.

Yet another important question when it comes to gene-tampering is how might it be misused?

For instance, might it be used for augmentation rather than treating disease? Potentially, using germline gene editing, you might increase the intelligence or eventual height of an as yet unborn child.

Some uses of genetic manipulation are already illegal in the U.S., but not everywhere.

A controversial possibility is xenotransplantation – genetically altering other animals so their organs can be used in humans. Pigs are one viable option and scientists are working on it. They are trying to modify the pig genome so the human body won’t see it as foreign.

Gene drives could introduce toxic genes into a species such as the mosquito that causes malaria, or the one carrying the Zika virus, killing off the entire population.

Gene editing is being studied to treat Sickle Cell Disease, hemophilia, and protection of cells from HIV infection.

And genetically modified crops are already helping battle world hunger but also creating considerable public debate.