A new study shows how new research can help with heart surgery
Researchers have shown that using a gene therapy to treat an inherited heart defect can improve a patient’s heart function.
Researchers say the gene therapy can be used to treat heart valve prolapse, which causes irregular heartbeats and can lead to a heart attack or stroke.
The study, published online in the journal Nature Medicine, was led by researchers at University College London, with help from the European Commission and National Institutes of Health.
The researchers say they found the gene-editing technique could be used by the next generation of heart transplants.
“It is the first time that we have been able to identify the gene that causes prolapse in patients with heart failure, and it may offer hope for future cardiac transplants,” lead author Dr Andrew J. Burt said in a statement.
“Our findings may also be useful for developing new gene therapies for heart failure that are safe, effective and affordable for patients.”
Burt and his colleagues looked at the gene known as CRISPR-Cas9, which was discovered in 2001 by scientists in France.
It is a complex protein that helps break DNA and copy its instructions into a specific sequence.
“With the CRISP-Cas 9 gene we identified the gene responsible for prolapse and we were able to show that this gene can be targeted in order to restore normal heart function,” said Burt.
“We used the same gene therapy approach to develop a gene that targets the protein CRISTR, which is involved in regulating the expression of genes and proteins in the heart, and this gene is also known to be involved in prolapse.”
The researchers injected the CRESPR-CISTR gene into healthy mice with heart defects, then switched it to a mouse model of heart disease.
“Mice that received CRISCR-CAS9 had improved cardiac function in response to the heart transplant treatment, which could be attributed to improved cardiac tissue and heart-related gene expression,” said Dr Benoit Baudry, a co-author of the study.
The gene therapy is being tested in mice in an effort to create a more effective version for people.
Previous research has shown that gene therapy in people has a number of benefits, including improving blood pressure, reducing heart disease risk, and lowering blood sugar.
However, it has been difficult to find a gene-based therapy that works in people, which has hindered development.
“Until now, we have only been able the CRISE gene to be used in humans for a very limited time,” Burt added.
“The CRISPAR gene has now shown promise in terms of being able to treat some of the other diseases associated with heart disease, including atrial fibrillation, angina and coronary artery disease.”
Dr Burt and other researchers have also studied gene therapy for other conditions, such as cystic fibrosis and multiple sclerosis.
The team hopes to find new gene-therapy therapies for some of these conditions, which would allow patients to live with their heart defects.
“While gene therapy might be a very promising treatment, it is still a long way from being able with all the complications associated with it,” said the lead author of the paper, Dr E. Scott McArthur, who is a clinical scientist at University of Liverpool.
“However, these findings show that gene-targeted gene therapy offers the potential to deliver a cure to patients with congenital heart failure.”