How gene-editing technology could help lower cholesterol
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How gene-editing technology could help lower cholesterol

Boston doctor explains how gene-editing technology could help lower cholesterol

FIVE AT 535 ON YOUR HEALTH TONIGHT. A SMALL STUDY SHOWS BIG PROMISE USING GENE EDITING TECHNOLOGY TO LOWER CHOLESTEROL. RESEARCHERS SAY THE EXPERIMENTAL TREATMENT HAS THE POTENTIAL TO HELP MILLIONS. HERE TO EXPLAIN IS DR. ALI RAJA, DEPUTY CHAIR OF THE DEPARTMENT OF EMERGENCY MEDICINE AT MASS GENERAL. GREAT TO SEE YOU, DOCTOR RAJA. THANKS FOR BEING HERE. YOU TOO, ERIKA. ALL RIGHT. DR. RAJA, LET’S START WITH THE STUDY. IT’S VERY SMALL. WE’RE TALKING JUST TEN PATIENTS, AND EACH ONE HAD A GENETIC ABNORMALITY THAT CAUSES VERY HIGH LEVELS OF CHOLESTEROL IN THE BLOOD. AND A VERY HIGH RISK FOR HEART DISEASE AT AN EARLY AGE. SO HOW DOES THIS GENE EDITING TREATMENT HELP? IT’S KIND OF AMAZING. BEN SO THIS GENE EDITING TREATMENT CONSISTS OF THESE NANOPARTICLES THAT ENTER LIVER CELLS AND THEY EDIT A SPECIFIC GENE THAT NORMALLY LEADS TO HEART ATTACKS AND STROKES, BUT INSTEAD THEY GO IN, THEY EDIT THE DNA. THEY TURN OFF THAT GENE. AND IN THEORY, IT WORKS FOR THE REST OF THESE PATIENTS LIVES. AND IN THIS TRIAL, AT LEAST, YOU SAID IT WAS ONLY TEN PATIENTS. THAT’S RIGHT. BUT THE THREE PATIENTS WHO RECEIVED THE HIGHEST DOSES OF THIS MEDICATION HAD THEIR CHOLESTEROL. FALL BETWEEN 40 AND 55%. WOW. AMAZING. OKAY. WE KNOW MOST PEOPLE DEVELOP HIGH CHOLESTEROL AND HEART DISEASE FOR OTHER REASONS. LIFESTYLE CHOICES. SO HOW COULD THIS RESEARCH MAYBE APPLY TO THEM? YOU WRITE ERIKA THE TEN PATIENTS. THIS STUDY ALL HAD THIS REALLY RARE DISEASE. SO WE DON’T KNOW IF THIS WILL WORK WITH OTHER PATIENTS. WE ALSO DON’T KNOW HOW SAFE IT IS, BOTH IN THE LONG TERM FOR THESE PATIENTS AS WELL AS FOR OTHER PATIENTS. BUT THE POTENTIAL OF THE CONCEPT OF BEING ABLE TO LOWER CHOLESTEROL FOREVER FOR PATIENTS WITHOUT THEM HAVING TO TAKE OTHER MEDICATION, THAT COULD REALLY BE A GAME CHANGER FOR LOTS AND LOTS OF PEOPLE. ALL RIGHT. DR. RAJA, AS YOU KNOW, THE FDA IS GOING TO DECIDE WITHIN WEEKS. NOW WHETHER TO APPROVE A GENE EDITING TREATMENT FOR SICKLE CELL DISEASE. SOME SPECIALISTS SAY IT’S A CURE, BUT THERE ARE SOME SAFETY CONCERNS, AS YOU MENTIONED, WITH THIS TYPE OF TECHNOLOGY OVER THE LONG TERM. SO WHAT DO PATIENTS NEED TO KNOW? YEAH, BEN THIS TREATMENT IS JUST AS AMAZING. IT’S JUST MORE COMPLICATED. SO THIS ONE IS CALLED X-CELL. AND WHAT IT DOES IS IT ACTUALLY EDITS PATIENT’S OWN BONE MARROW IN A WAY THAT DECREASES THE SYMPTOMS OF SICKLE CELL DISEASE. BUT THIS ONE ACTUALLY REQUIRES THAT DOCTORS COLLECT THE PATIENT’S BONE MARROW, TREAT IT, AND THEN IT REQUIRES A ROUND OF CHEMOTHERAPY SO THAT THE PATIENT’S BODY ACTUALLY ACCEPTS THAT MODIFIED BONE MARROW BACK. SO IT’S A LOT MORE COMPLICATED WITH A LOT OF RISKS. BUT THE DATA LOOK REALLY PROMISING AND SICKLE CELL CAN BE SUCH A DEVASTATING DISEASE. SO THE BENEFITS PROBABLY DO OUTWEIGH THE RISKS FOR MOST PATIENTS. AMAZIN

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Boston doctor explains how gene-editing technology could help lower cholesterol

Researchers say the experimental treatment has the potential to help millions.

Researchers say the experimental treatment has the potential to help millions.

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