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Gene Editing Breakthrough: How Far Are We From Fixing And Designing Babies? – Forbes

Gene editing technology allowed scientists to remove a mutation from human embryos. How far are custom-designed babies? (Photo: Shutterstock)

How far is our society from genetically modifying people like Microsoft Word documents? A recent publication in Nature described the way a team of scientists used the CRISPR-Cas9 gene editing system to cut out a heart disease-causing mutation from the MYBPC3 gene in early stage human embryos and then paste in a normal version of the genetic sequence. This is yet another example of scientists editing the genes of human embryos after researchers in China first reported similar success for a different mutation in 2015. Being able to essentially use Control+X and Control+V “buttons” on human embryos conjures up not only visions of searching and cleaning embryos for dangerous genetic mutations but also nightmares of a Utopian Brave New World or Gattaca in which babies are genetically engineered for their gender, appearance, and abilities. There’s also the horror movie settings such as The FlySharktopus, Leviathan, Resident Evil, and The Simpsons in which experimenting with genetic material ends up creating horrible consequences. No, gene editing is not the same as editing a Word document.

Now, I usually don’t use horror or science fiction movies to better understand the consequences of science, especially a movie that involves a half-shark and half-octopus creature attacking a bikini-clad woman on a bungy cord. However, movies that portray the fictional consequences of gene editing do highlight the fact that genes and their interaction with the environment over time are extremely complex, gene editing human embryos could have unintended consequences, and gene editing human embryos brings ethical concerns. Two years ago the U.S. Congress banned clinical trials that use gene editing on human embryos. However, continuing such a ban will prevent scientists from further testing whether gene editing human embryos can indeed prevent disease. Gene editing an embryo in a lab is one thing. Determining whether this will actually improve health is a whole different ballgame.

However, should the ban eventually be lifted, current clinical trials designs may not be adequate to really test this technology. To really fully explore the longer term effects, both good and bad, of gene editing in a manner that allays ethical concerns may require re-thinking how clinical trials are designed and run. In fact, gene editing could further expose some of the existing flaws of clinical trials and even help “edit” how they are designed and run in general.

CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeat and is pronounced like what you do to bread when your leave it in the toaster longer. The CRISPR-Ca9 gene editing system consists of a protein called Ca-9, which functions like a pair of scissors, and an attached piece of RNA that matches the DNA that you want to alter. After being injected into a cell, the RNA helps find the DNA segment and the Ca-9 snips out the DNA. Either the cell will naturally fix and replace this now missing DNA or scientists can include a replacement DNA strand. It sounds simple just like editing a Word document, replacing a tire, or changing an article of clothing. Here biologist Neville Sanjana explains CRISPR to a 7 year-old, a 14 year-old, a college student, a grad student and a CRISPR expert, not that the 5 categories are always completely mutually exclusive:

Such technology has widespread potential applications. I talked to Jennifer Doudna, PhD., Professor of Chemistry and Molecular and Cell biology at the University of California, Berkeley, and Emmanuelle Charpentier, PhD, Director at the Max Planck Institute for Infection Biology in Berlin, when they received the prestigious Japan Prize in April of this year for their roles in inventing the CRISPR/Cas9 gene editing system. (Feng Zhang, PhD, Professor of Neuroscience at the Massachusetts Institute of Technology has also been credited with inventing the technology). Both Doudna and Charpentier described how they developed the CRISPR/Cas9 gene editing system after studying how bacteria identify and chop up the DNA of invading viruses. They also described how such a system could eventually help prevent and treat diseases in humans and animals and improve crops but also recognized the dangers of using this scientific breakthrough to custom design and “manufacture” babies.

Japan Prize Foundation

Dr. Jennifer Doudna (second from left) and Dr. Emmanuelle Charpentier (center) received the Japan Prize this year for their role in developing the CRISPR-Ca9 gene editing system. (Photo courtesy of the Japan Prize).

This latest study (whose lead author was Hong Ma and senior author was Shoukhrat Mitalipov, both from the Oregon Health and Science University) demonstrated how CRISPR-Ca9 can cut out a genetic mutation responsible for hypertrophic cardiomyopathy and replace it with a normal genetic sequence. The mutation is one but not the only possible cause of hypertrophic cardiomyopathy, a condition in which the heat muscle grows abnormally thick, potentially disrupting the heart’s electrical system. Basketball players Reggie Lewis of the Boston Celtics and Hank Gathers of Loyola Marymount University suffered fatal heart rhythms from such a disruption. For this study, since the U.S. Congress has so far banned scientists from using CRISPR to edit embryos, the team grew the human embryos for only 3 days before discarding them. Although the gene editing system was not perfect in removing the mutation, it did seem to work in the majority of embryos. As the New York Times reported, of 54 embryos generated, 36 embryos were completely mutation free (versus half of the embryos, the rate at which the mutation is passed down to children under normal circumstances) and 13 more embryos had some cells but not all free of the mutation. One fear of gene editing is accidentally creating other unwanted mutations, but according to the Nature report, no other mutations appeared to result. As Emily Mullin reported for the MIT Technology Review, in a press conference call, Mitalipov explained, “With this particular mutation, we’ve already done the groundwork, so we’re probably much closer to clinical applications. Clinical trials would mean actually implanting some of these embryos with the goal of establishing pregnancy and monitoring births of children and hopefully following up with children.”

Gene Editing Breakthrough: How Far Are We From Fixing And Designing Babies? – Forbes

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