Gene editing breakthrough allows scientists to repair mutation


TED SIMONS: SCIENTISTS HAVE SUCCESSFULLY MANIPULATED GENES IN HUMAN EMBRYOS TO REPAIR A MUTATION THAT CAUSES SICKNESS. THE RESULT OF THE GENE EDITING: A HEALTHY EMBRYO. HERE WITH INSIGHT INTO ALL THIS IS DR. JAMES COLLINS, ASU'S VIRGINIA M. ULLMAN PROFESSOR OF NATURAL HISTORY AND THE ENVIRONMENT. GOOD TO SEE YOU BACK AGAIN.

DR. JAMES COLLINS: GOOD TO BE BACK AGAIN.

TED SIMONS: GENES EDITED IN HUMAN EMBRYOS TO REPAIR MUTATIONS?

DR. JAMES COLLINS: SURE. GENES EDITED IN HUMAN EGGS THAT GO ON TO DEVELOP INTO EMBRYO. IT WAS STOPPED BEFORE IT GETS TO A POINT WHERE IMPLANTATION WOULD BE POSSIBLE SO, YES, IT WAS A FASCINATING BIT OF TECHNOLOGY, EDITING THESE GENES THAT THEN GO INTO THE EGGS.

TED SIMONS: THE TEST WAS A COMMON HEART CONDITION THAT LEADS TO SUDDEN DEATH. SOUNDS LIKE THAT CONDITION GOES AWAY.

TED SIMONS: IT DOES. WE HAVE MOM AND DAD. WE ARE GETTING GENES FROM MOM. GENES FROM DAD. THEY COME TOGETHER AND DEVELOP INTO A YOUNGSTER. WITH THIS PARTICULAR TECHNIQUE, THE EXPERIMENT THEY DID, THEY HAD AN EGG. THEY HAD THE EGG WITH MOM'S DNA IN IT. THEY INJECTED SPERM, SO THAT WAS DAD'S DNA GOING INTO THE EGG. IT BORE A MUTATION, A DANGEROUS MUTATION THAT COULD CAUSE HEART FAILURE AS YOU SAID. THE OTHER THING THEY DID WAS THEY INJECTED A CHEMICAL CALLED CRISPR, AND THEY WERE ABLE TO THROUGH THIS PROCESS, NEUTRALIZE THE GENE IN DAD THAT WOULD HAVE CAUSED THE HEART PROBLEM, AND WOUND UP WITH A PERFECTLY GOOD DEVELOPING EGG.

TED SIMONS: MY GOODNESS. OKAY, SO PERFECTLY GOOD THERE. WHAT ABOUT THE DESCENDANTS IF THAT BECOMES A LITTLE PERSON NOW, A BIG PERSON, HAS CHILDREN, WILL THOSE DESCENDANTS STILL NEED THE MODIFICATION OR DO WE START ANEW?

DR. JAMES COLLINS: BECAUSE OF THE TIMING OF WHEN THE EXPERIMENT WAS DONE AND THE MANIPULATION WAS DONE IN THE BEGINNING OF DEVELOPMENT, THAT MEANS THAT THE EMBRYO WOULD DEVELOP AND HAVE THIS REPAIR IN ALL OF ITS CELLS, WHICH MEANS IF AN INDIVIDUAL WERE ALLOWED TO DEVELOP, THE CHILDREN WOULD HAVE THE GENE THAT WOULD BE NOT THE DAMAGED ONE. IT WOULD BE A REPAIRED ONE.

TED SIMONS: SO WE ARE BASICALLY -- ARE WE TALKING ABOUT GENERATIONS FROM NOW, MANY MUTATIONS WOULD BE ELIMINATED LEADING TO CERTAIN CONDITIONS?

DR. JAMES COLLINS: YOU WANT TO BE CAREFUL BECAUSE THEY PICKED A GENE WITH A MAJOR EFFECT, AND A SINGLE GENE WITH A MAJOR EFFECT. IF WE THINK ABOUT TRAITS SUCH AS HEIGHT OR INTELLIGENCE, HUNDREDS OF THOUSANDS OF GENES ARE INVOLVED. FURTHERMORE, THEY INTERACT IN THE ENVIRONMENT. SO THAT TYPE OF THING IS A LONG, LONG WAY.

DR. JAMES COLLINS: SO THERE ARE ETHICAL CONCERNS.

DR. JAMES COLLINS: MANY ETHICAL CONCERNS. YOU HAVE TO DO A LOT OF WORK TO ADDRESS THE ETHICAL CONCERNS AND YOU HAVE TO START AT THE BEGINNING TO DO THESE TYPE OF MANIPULATIONS BECAUSE THERE ARE INDIVIDUALS WHO ARE OPPOSED TO IT FROM THE START.

TED SIMONS: THINGS LIKE SICKLE CELL ANEMIA, CYSTIC FIBROSIS, HUNTINGTON'S DISEASE, BREAST CANCER, THOSE THINGS COULD BE AFFECTED.

DR. JAMES COLLINS: THEY COULD BE AFFECTED BECAUSE THEY ARE ALSO DISEASES CAUSED BY A MAJOR DOMINANT EFFECT. IN THIS CASE, YOU COULD GO AFTER THEM WITH THIS SORT OF MANIPULATION.

TED SIMONS: BUT THERE IS AN ETHICAL TYPE OF ISSUE FOR THOSE WHO COULD AFFORD THIS TYPE OF GENE MODIFICATION. IS THAT SOMETHING TO WATCH OUT FOR?

DR. JAMES COLLINS: DEFINITELY. THESE KINDS OF MANIPULATIONS ARE EXPENSIVE. YOU DON'T WANT IT AVAILABLE TO A SELECT SEGMENT OF SOCIETY. YOU WOULD LIKE IT TO BE AVAILABLE TO EVERYONE.

TED SIMONS: GREATER INTELLIGENCE, GREATER ATHLETICISM, THESE SORT OF THINGS, YOU CAN ONLY GO SO FAR WITH GENE EDITS.

DR. JAMES COLLINS: AT THIS POINT, THAT’S RIGHT. IT'S A LARGE NUMBER OF GENES AND THE SORT OF INTERACTIONS INVOLVED, IT WOULD TAKE IMMENSE AMOUNT OF CHANGES. IN ORDER TO PREDICT THE OUTCOMES, IT WOULD BE VERY, VERY HARD.

TED SIMONS: LAST QUESTION, HOW BIG OF A DEAL IS THIS?

DR. JAMES COLLINS: IT'S A BIG DEAL. IT'S ONE MORE STEP DOWN THE ROAD OF BEING ABLE TO USE TECHNOLOGY IN A VARIETY OF WAYS. WE USE TECHNOLOGY ALL SORTS OF WAYS TO MAKE LIFE BETTER FOR HUMAN BEINGS. THIS IS ONE MORE STEP DOWN THAT ROAD IN A FASCINATING WAY TO MANIPULATING GENES AND ORGANISMS.

TED SIMONS: IT'S A FASCINATING STORY. THANK YOU FOR CLEARING THAT UP FOR US. THURSDAY ON "ARIZONA HORIZON," HOW BEST TO EXPERIENCE THE UPCOMING SOLAR ECLIPSE. AND AN INVESTIGATION BY STUDENT JOURNALISTS INTO THE STATE'S WATER SYSTEM. THOSE STORIES ON THE NEXT "ARIZONA HORIZON." THAT IS IT FOR NOW. I'M TED SIMONS. THANK YOU SO MUCH FOR JOINING US. YOU HAVE A GREAT EVENING.

For the first time, scientists have edited genes in human embryos to repair a mutation that causes sickness, ending up with a healthy embryo. We’ll discuss this scientific breakthrough.

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