Could one-time gene-editing injections cure genetic heart disease?

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Silence certain genes could become the cure for inherited heart disease, according to new research. Andrew Brookes/Getty Images
  • Cardiomyopathies are disorders that impair the function of the heart, and many cases are genetic in origin.
  • Experts are exploring the use of gene therapies to treat cardiomyopathies.
  • A research team called CureHeart recently received a £30 million grant from the British Heart Foundation to fuel new research into genetic medical treatments to help people with cardiomyopathies.
  • Researchers will work on the development of an injectable remedy that works by silencing or modifying certain genes.

Heart disease can take many forms and develop based on components such as genetics, environmental factors, and lifestyle. Disorders that affect the muscles of the heart, or cardiomyopathies, may have genetic origins, which has fueled research into possible genetic treatments to cure them.

The research team CureHeart recently received a £30 million grant from the British Heart Foundation (BHF) after winning the BHF competition, the Big Beat Challenge. The money will be used to advance the group’s progress in genetic treatments for cardiomyopathies.

Cardiomyopathy is an umbrella term for all disorders affecting the muscles of the heart. When a person has cardiomyopathy, it may be more difficult for the heart to pump blood throughout the body efficiently.

People with cardiomyopathy can experience a variety of symptoms ranging from mild to severe. There are many types of cardiomyopathy. Heart muscles can thicken, become stiff, or even widen, depending on the type. Cardiomyopathy can cause serious heart problemsincluding abnormal heart rhythms, heart failure, cardiac arrest and stroke.

Dr Edo Pazcardiologist and medical vice president of digital care app K Health, which is not affiliated with CureHeart, explained to Medical News Today:

“Cardiomyopathies are diseases of the heart muscle. They can be extremely serious, as patients can develop congestive heart failure and irregular heart rhythms that can lead to sudden cardiac death. There are many types of cardiomyopathy and treatment varies depending on the type and/or cause.

Dr. Paz also discussed current treatments.

“[I]In most cases of cardiomyopathy, there are a number of medications that can improve the patient’s symptoms, improve or prevent worsening heart muscle function, and improve survival. Other treatments that may apply to some cardiomyopathies include surgery and/or placement of an implantable cardioverter defibrillator. In severe cases, patients may undergo heart transplantation,” he said.

Sometimes cardiomyopathy can have a genetic origin. When doctors know that a person has specific genetic variants associated with the disease, they can be proactive about monitoring and treatment options. However, unlocking the genetic causes of cardiomyopathy could also hold the key to developing new treatments.

As part of its efforts to advance the treatment of heart disease, the BHF organizes the global competition, the Big Beat Challenge. Different cardiology research teams are vying to win £30m to fund their research, presenting their ideas to a panel of patients and the public, as well as a panel of researchers and healthcare professionals. The final decision was taken by an international advisory committee made up of personalities from science and medicine.

The winner of the Big Beat Challenge was the research team, CureHeartwhich focused on genetic treatments for cardiomyopathies.

They explain on their website that their goal is to “develop gene therapies that precisely correct faulty genes in the heart itself, thereby providing a new treatment for, and ultimately a cure for, cardiomyopathies.”

“Around 1 in 250 people are affected by genetic diseases of the heart muscle, collectively known as cardiomyopathy. Around 260,000 people in the UK and 1.5 million in the US are affected. Many are unaware they may be at risk.
— Researchers from the CureHeart team

Researchers want to focus their efforts on correcting and turning off genes that contribute to cardiomyopathies.

As part of their work, they identified cardiomyopathy genes and how they cause cardiomyopathy. They also worked on how to deliver the genetic medicine so that it hits the target heart muscle cells.

The CureHeart team is excited about how their research could help people with genetic cardiomyopathies. Yet they also note that their research could extend to other areas, such as the treatment of heart failure. They note that genetics may play a role in heart failure, and addressing the genetic root cause could be very beneficial.

Developing cures for cardiomyopathies could impact the lives of many people, including children affected by cardiomyopathies.

In a recent Press release, Professor Sir Nileshthe medical director of the British Heart Foundation, said the team’s work had great potential.

“Once successful, the same gene-editing innovations could be used to treat a range of common heart conditions in which genetic defects play a major role. This would have a transformational impact and offer hope to the thousands families around the world affected by these devastating diseases,” he says.

“This is a watershed moment for cardiovascular medicine. Not only could CureHeart be the creator of the first cure for inherited heart muscle diseases by tackling killer genes that cross family trees, but it could also usher in a new era of precision cardiology.
—Professor Sir Nilesh

Professor Hugh Watkinsa leading member of the CureHeart research team and Professor of Cardiovascular Medicine at the British Heart Foundation, said he was excited about the funding and development of research in this area.

“I am excited about the BHF Big Beat Challenge because the magnitude of the award has recruited world-class scientists, including some who have not previously worked in heart disease,” he said. MNT.

“We believe we have everything we need now to discover how best to use gene therapies to solve the fundamental problem of inherited heart muscle diseases and start creating cures where none exist in the past. current time,” he added.