For the first time, the new program aims to cure genetic heart diseases

  • New research venture could cure genetic heart disease.
  • The global team of experts assembled for the project expects to be ready to begin testing gene therapy in clinical trials within the next five years.
  • New gene therapy could cure many other genetic diseases.

Genetic heart disease can be treated for the first time in the world.

A global team of experts from the UK, US and Singapore are joining forces for the CureHeart Project to develop an effective genetic therapy to treat cardiomyopathies, or diseases of the heart muscle that make it difficult for the heart to pump blood to the rest of the body. . After the team recently received an estimated $37 million grant from the British Heart Foundation to go towards life-saving research, the venture is expected to cure countless sufferers.

Scientists are using precise genetic techniques called core and core editing in the heart to develop and test the first cures for inherited heart muscle disorders. deleterious mutations). Although researchers have not yet reached human trials, the treatment is promising because of its success in animal trials.

“For decades, we’ve been hoping to cure heart disease. Most heart disease is currently incurable, but simply manageable,” said Richard Wright, MD, a cardiologist at Providence St. John’s Health Center in Santa Monica, California. prevention. “At least in this array of unusual diseases, there’s a chance that a specific gene is abnormal, and if we can identify it, theoretically, we can go inside the cell and solve the underlying problem in these people. and heals them.’

All people with inherited heart muscle disorders, known as genetic cardiomyopathies, have a 50/50 chance of passing the faulty gene to each of their children. And often several members of the same family get heart disease, need a heart transplant, or die at a young age. This was reported by Oxford University professor Hugh Watkins and the chief researcher of the CureHeart project. The Guardian cardiomyopathies are “really common” and affect one in every 250 people worldwide.

“This is a once-in-a-lifetime opportunity to save our families from sudden death, heart disease and the need for a heart transplant,” Watkins said. The Guardian. “After 30 years of research, we have discovered many genes and specific genetic defects responsible for various cardiomyopathies and how they work. We believe we will have gene therapy to start clinical trials in the next five years.”

About half of the genes that work to make proteins in the heart, and thousands of errors in the genetic code that cause the heart to become too weak or the heart muscle to be too thick, can lead to many health problems. issues. These mutations are one of the most common causes of heart failure, says Dr. Wright. “If this treatment is successful, you could quickly reprogram the genes to make normal proteins instead of the abnormal proteins inside the heart, which would restore the heart’s function back to normal.”

Just because you inherit a “bad” gene doesn’t necessarily mean you’ll get the disease. Some people carry these mutations or “bad genes” throughout their lives and never show any symptoms. However, in some specific cases, we know for sure that the disease will occur eventually in life, in which case we want to prevent it from ever happening. This cure can be distributed to patients with cardiomyopathy at any time in their lives, before or after the onset of the disease.

Bottom line

This new research has the potential to cure not only heart disease but also many other genetic diseases. “Genetic blood disorders like sickle cell anemia, or brain disorders like Huntington’s or chorea, are diseases where we know what the problem is, but we don’t have the tools to fix the problem,” says Dr. Wright.

How can you go into a cell and edit the genetic code without causing more damage? In the past, scientists used viruses to carry information into cells, which had potential side effects, but now they can do it directly and completely destroy the carrier, which may be much safer, Dr. Wright.

However, there are still many aspects to this treatment if and when recovery becomes easier. Although the research team consists of the best scientists from around the world, there is still much to be done. Dr. Now, Wright says, “they finally have arrows that they can use to attack this problem.”

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