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Through genetic modification, humans are taking the reins from Mother Nature, making huge strides in fields such as human nutrition and health. Editing genes can help parents with genetic diseases bear healthy babies. Such technology can also lead to the creation of higher-yielding crops and cure cancer with immune cells that are injected into the human body. As our speakers noted, this work also raises the risk of unintended consequences and bears thoughtful consideration by a wide set of stakeholders.

The biggest recent advance accelerating the pace of biotech research is known as CRISPR (clustered regularly interspaced short palindromic repeat), according to Edison Liu, president and CEO of The Jackson Laboratory, which focuses on genomic research. Using CRISPR, scientists target and modify specific genetic sequences in our DNA. By changing the sequences, scientists using CRISPR can accomplish incredible things: cure many genetic diseases, or modify plants to increase production or resistance to disease or pests.

CRISPR has dramatically boosted the efficiency of genetic modification while reducing the cost by about seven times, according to Liu. "This speed, precision and comprehensiveness has never been seen before,” he said. "What is exciting is that the technology has the promise of absolute precision."

Scientists could use CRISPR technology to target human cells that carry mutated genes, such as cells infected with viruses similar to HIV, Liu said. Already, the technology has been used to genetically engineer mosquitoes, proving extremely effective in combating mosquito-borne illnesses, such as malaria. Still, Liu noted the potential negative consequences from annihilating an entire species, even one harmful to humans, such as mosquitoes.

Andrew Hack, chief financial officer at biotech startup Editas Medicine, said his firm plans to use CRISPR technology to perform precise genetic modification to treat a wide variety of human diseases. For example, the company, which raised $100 million in an initial public offering and $120 million in a Series B round led by Bill Gates's advisor, aims through genetic research to find techniques for restoring vision for people who suffer from a form of leber congenital amaurosis (LCA), a disorder of the retina that can lead to severe visual impairment or blindness.

Brown Advisory's NOW 2016 | GENOMES AND GENE EDITING: A NEW CHAPTER IN MEDICINE
Many of the world’s most devastating diseases—cancer, cystic fibrosis, muscular dystrophy and others—can be traced back to genetic markers and genetic errors. The medical and scientific communities have long struggled to address these root causes, and incredible recent advances in genomics and genetic programming offer new hope to sufferers of conditions once considered incurable. Dr. Edward Liu, CEO of The Jackson Laboratory, one of the world’s leading genomics researchers, is joined by Andrew Hack, M.D., Ph.D., Chief Financial Officer, Editas Medicine, to provide a vision of a future in which we can attack disease at the genetic source—a future that is not as far away as we might imagine.Editas also plans to collaborate with Juno Therapeutics, a leading biopharmaceutical company focused on re-engaging the body's immune system to combat cancer. Editas' mission is to translate its genome editing technology into a new class of drugs that enable precise molecular modification to treat a broad range of diseases at the genetic level.

Liu and Hack said that the ethical challenges posed by editing of human genes is limited to some degree because such activity focuses on somatic cells that will not be passed on to future generations. Indeed, a core aim of gene editing is to treat human diseases without altering the genome, they said. Some scientists believe that current technology could also help parents who are carriers of genetic diseases to have healthy babies.

The public conversation over genetic engineering should focus on the point of view of the patient, Hack said, noting that biomedical companies are devoted to treating patients, not altering genetic fate or changing the genetic traits of future generations.

Liu said the discussion about the future of bioengineering should occur throughout society and not just among scientists. "I don't believe that with powerful tools like this, it can only rest in the hands of scientists," he said. "It has to be a societal discussion and one that has both thoughtful governance as well as controls within the system."

 

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