Scientists'rewrite' DNA in order to obtain the first treatment for genetic heart diseases

Scientists'rewrite' DNA in order to obtain the first treatment for genetic heart diseases ...

Heart disease can take many forms and develop depending on factors such as genetics, environmental factors, and lifestyle. Disorders that impact the heart muscles, or cardiomyopathies, can have genetic origins, which has fueled interest in possible genetic therapies to treat them.

After winning the BHFs Big Beat Challenge, the research team CureHeart was recently awarded a 30 million grant from the British Heart Foundation. The money will be used to support the group's research efforts in genetic cardiomyopathies.

Cardiomyopathy and treatment options

Cardiomyopathy is a broad term that encompasses all disorders that affect the heart's muscles. It may be more difficult for the heart to pump blood efficiently throughout the body when someone has cardiomyopathy.

People with cardiomyopathy may have a wide range of symptoms, from mild to severe. Depending on the type, the heart muscles may contract, become rigid, or even expanded. Cardiomyopathy may result in severe cardiac problems, including cardiac arrest, and stroke.

Dr. Edo Paz, a cardiologist and medical vice president at K Health, a digital care app that isn't affiliated with CureHeart, told Medical News Today:

Cardiomyopathies are cardiac injuries that are quite common. They can be quite severe, as patients may develop congestive heart failure and irregular heart rhythms that may result in sudden cardiac death.

Dr. Paz also touched on current therapies.

There are a number of medications that can improve patient symptoms, improve or prevent worsening of cardiac muscle function, and improve survival in most cases of cardiomyopathy, according to the doctor. Other treatments that may apply to certain cardiomyopathies include surgery and/or the placement of an implantable cardioverter-defibrillator in severe instances.

Cardiomyopathy may be caused by a genetic mutation. Doctors may be proactive when it comes to monitoring and treatment options. However, discovering the genetic causes of cardiomyopathy may also be the key to developing new treatments.

Generating and reversing them is a straightforward process.

The British Heart Foundation has organized the Big Beat Challenge, a global competition for heart disease research. Different cardiology research teams compete for the chance to win 30 million to support their research, before before a patient and public panel, and to a panel of scientists and medical professionals. The final decision was made by an international advisory committee that included leading scientists and physicians.

CureHeart, a research group focusing on genetic therapies for cardiomyopathies, was the winner of the Big Beat Challenge.

On their website, they state that their intention is to develop genetic treatments that correct precisely the abnormal genes in the heart, thus introducing a new therapy for, and ultimately treating, cardiomyopathies.

Genetic heart muscle diseases, collectively called cardiomyopathy, affect 1 in every 250 people; many are unaware that they are at danger. Researchers from the CureHeart team examined how 260,000 people in the United Kingdom and 1.5 million in the United States have been affected.

The researchers want to concentrate their efforts on correcting and shutting off the genes that contribute to cardiomyopathies.

As part of their research, they have identified cardiomyopathy disease genes and how they cause cardiomyopathy. They have also studied how to deliver genetic medicine so that it arrives at the heart muscle cells' target.

The field of research is moving forward.

The CureHeart team is excited about how their research might assist those with genetic cardiomyopathies. However, they also note that their research may extend to other areas, such as medicine for heart failure.

The development of cardiomyopathies cures might be beneficial to many people, including children.

Professor Sir Nilesh, the British Heart Foundation's medical director, said the teams' work had enormous potential in a recent press release.

Once successful, these gene editing methods may be applied to a wide spectrum of common heart conditions where genetic deficiencies play a significant role. This would have a transformative impact and provide hope to the thousands of families affected by these devastating diseases worldwide, according to the researcher.

CureHeart might be the first to develop a cure for inherited heart muscle diseases by tackling killer genes that go through family tree roots, but it might also usher in a new era of precision cardiology. Prof. Nilesh

Prof. Hugh Watkins, who is a senior member of the CureHeart research group and is a professor of cardiovascular medicine at the British Heart Foundation, said he is excited about the funding and research in this area.

The BHF's Big Beat Challenge has me greatly impressed because of the scope of the prize, which allowed for the use of some truly world-class scientists, some of whom were not previously involved in the field of heart disease, according to MNT.

He said, "We believe we have everything we need now to discover how to best utilize genetic treatments to address the fundamental problem in inherited heart muscle disorders," and begin to develop treatments where none exist at the moment.

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