New program aims to treat genetic heart disease for the first time

  • A new research project could lead to a treatment for genetic heart disease.
  • The global team of experts assembled for this project expects the gene therapy to be ready to begin testing in clinical trials in the next five years.
  • New gene therapy could lead to treatments for a plethora of other genetic diseases.

The first-ever treatment for genetic heart disease appears to be within reach.

A global team of experts from the UK, US and Singapore are uniting for Project CureHeart to develop effective gene therapies for cardiomyopathy, or heart muscle diseases that make it difficult for the heart to pump blood to the rest of the body. . This project is expected to treat countless affected people after the team recently won a grant of nearly $37 million from the British Heart Foundation to go towards life-saving research.

Researchers will use precise genetic techniques, called basic and essential modification, in the heart for the first time to design and test the first treatment for inherited heart muscle disease, with the goal of turning off the faulty genes (so that they no longer express harmful mutations). While the researchers are not yet in human trials, the treatment is promising, having already seen success in animal trials.

“For decades, we’ve hoped we could cure heart disease. Right now, most heart conditions are incurable, but only manageable,” says Richard Wright, MD, a cardiologist at Providence Saint John’s Health Center in Santa Monica, Calif. protection. “At least for this unusual group of disease conditions in which a particular gene is abnormal and we’ve been able to identify it, there’s a potential chance that, in theory, we can go straight into the cell and fix the underlying problem in these people and treat them.”

All those with hereditary cardiomyopathy, also known as hereditary cardiomyopathy, have a 50/50 risk of passing one defective gene to each of their children. Many members of the same family often develop heart failure, need a heart transplant, or die at a young age. Professor Hugh Watkins, of the University of Oxford and Principal Investigator of the CureHeart Project, told Watchman Cardiomyopathy was “really common”, affecting 1 in 250 people worldwide.

“This is our once-in-a-generation opportunity to relieve families’ ongoing anxiety about sudden death, heart failure and the potential need for a heart transplant,” Watkins said. Watchman. “After 30 years of research, we have discovered many specific genes and genetic defects responsible for different cardiomyopathy, and how they work. We believe we will have a gene therapy ready to begin testing in clinical trials in the next five years.”

About half the genes we turn on to make proteins in the heart, and there are thousands of errors in the genetic code that lead to a weak heart or too thick heart muscle, which can lead to a myriad of health problems. Dr. Wright says these mutations are one of the most common causes of heart failure. “If this treatment is successful, you can instead quickly reprogram the genes to make the proteins normal, rather than the abnormal proteins within the heart, causing the heart to reverse its dysfunction and return to normal.”

If you inherit a “bad” gene, this does not necessarily mean that you will end up with the disease. Some people carry these mutations or “bad genes” their whole lives and never show any disease. However, in some specific cases, we know for sure that the disease will eventually appear later in life – in which case, we may want to prevent it from happening at all. It is possible that this treatment will be distributed to those who suffer from cardiomyopathy at any time in their lives, before or after the onset of the condition itself.

bottom line

This ground-breaking research could mean a cure not only for heart disease but also for many other genetic diseases. Dr. says. I have seen.

So how do you get into the cell and correct the genetic code without causing more damage? Scientists used to use viruses to transmit information into the cell, which obviously has its potential side effects, but now they have the ability to do so directly and cut off the carrier altogether, which may be safer, according to Dr. Wright. .

But, there are still many aspects of this treatment to consider, if and when treatment becomes readily available. Although the research team consists of some of the leading scientists around the world, much remains to be done. Now Dr. Wright says, “They finally have arrows up their sleeve that they can use to attack this problem.”

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