Pushing the Boundaries of Science to Advance Human Health

Scientists say gene editing holds the key to curing a host of intractable diseases, including cystic fibrosis, HIV, cancer, and cataracts, to name a few. Policymakers fear that, if this key fell into the wrong hands, it could open a Pandora's box of dangers such as eugenics and performance enhancements on demand. Two Penn Integrates Knowledge professors who are experts in the field recently discussed these pressing issues with Dan Loney on Wharton Business Radio.

James W. Effron University Professor John Gearhart, who holds appointments in the Perelman School of Medicine (PSOM) and in the School of Veterinary Medicine and has served as director of Penn's Institute for Regenerative Medicine, discovered how to isolate and propagate the world's first human pluripotent stem cells in 1998.

David and Lyn Silfen University Professor Jonathan D. Moreno, who holds appointments in PSOM and the School of Arts & Sciences, serves as a senior advisor on the Presidential Commission for the Study of Bioethical Issues and recently returned from the International Summit on Human Gene Editing held in Washington D.C.

PIK Professors John Gearhart and Jonathan Moreno

Professors Gearhart and Moreno

Current Capabilities and Concerns

According to Moreno, there's always been a clear distinction between traditional gene therapy, which involves changing the DNA in a person's body cells, and germline engineering, which involves changing the DNA in the gametes that develop into a person. Recently, however, this distinction has been blurred by the unprecedented precision and efficiency of a new technology called gene editing, which enables scientists to change, replace, or delete a person's genes with the end goal of correcting mutations, damage, or defects.

Managing the Risks

Gene editing could dramatically improve the quality of life for people who suffer from medical conditions that range from debilitating to fatal—but along with these breakthroughs come risks: The therapy could go off-target, incurring unanticipated side effects (although this targeting is becoming more precise by the day). These benefits and risks are heightened in germline engineering, which could reduce susceptibility to certain diseases—or introduce rogue mutations—not just in a given patient, but in all of his or her descendants.

"The shadow of eugenics and trying to improve the human race" lurks over germline engineering, too, says Moreno, "but it's a whole lot harder than people think to do that."

The Marketplace and the Mosquito

According to Gearhart, somewhere between $40-80 billion has been invested world-wide in regenerative medicine research, and the resulting technologies promise to yield an exponentially greater return—in quantifiable ways, as well as having the undisputable benefit of eliminating susceptibilities to diseases and defects caused by genetic mutations.

Public health costs would plummet as a result of the elimination of deadly diseases. For example, a strain of mosquitoes with malaria-blocking genes has already been engineered, and when introduced into a population of wild mosquitoes, it could effectively wipe out the disease in that region.

Agriculture and food industries would expand as a result of longer growing seasons and resistance to pests and disease. Already, strawberries can be safely modified to resist frost, and salmon safely modified to grow twice as fast to meet a burgeoning market.

A Global Community of Scientists

According to Moreno, the scientific community has changed dramatically in the last 30-40 years. It now consists of a global network of researchers who have immediate access to the most cutting-edge work being done today. Scientists anywhere can see what leaders such as Gearhart have done and say, "'Well I wonder if I can just tweak it a little bit and get this result,'" he says.

Concern for the consequent breakneck pace of growth in the field of gene editing was one of the reasons the Summit in Washington D.C. was convened earlier this month, with members of the community recognizing the need to discuss the ethics of gene and germline editing in an international forum. Moreno details this discussion in his recent Scientific American article.

Navigating the Politics

Gearhart agrees that international summits such as these are essential. "But," he adds, "the concern here is one of governmental interference that could stifle a lot of good."

Gearhart has made upwards of 200 appearances in Washington D.C. alone to speak about research threatened by such interference, including stem cells, cloning, human embryo and fetal tissue research, germline modifications, gene therapy, human/animal chimeras, and regenerative medicine.

He says that developing prudent policy and keeping the public informed is crucial to removing fears about new technologies such as these. "And this is something that Penn is very good at, with its outstanding investigators, clinicians, and ethicists, paired with its commitment to addressing societal needs and concerns."

Professors Gearhart and Moreno

  • Text by Christina Cook
  • Photos by Scott Spitzer
  • Audio excerpts courtesy of Wharton Business Radio