Saturday January 18, 2020

Gene Therapy Can Help Correct Heart Rhythm Disorder

However, considerable research is still needed before a treatment can be arrived at that is suitable for human patients

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The study modified the levels of the protein encoded by a single gene known as GPR39.  Pixabay

A novel gene therapy uses an implanted LED device to reset a racing heart immediately and automatically, paving the way for pain-free treatment for patients with heart rhythm disorder.

The therapy detects fast arrhythmias in the atrium of a rat’s heart and sends a signal to a LED device placed near the heart.

“The flash of light from this LED then causes the heart to generate an electric current itself to halt the arrhythmia,” said lead investigator Daniel Pijnappels at Leiden University in the Netherlands.

“This is made possible by using gene therapy to introduce specific light-sensitive proteins into the heart. This restores the heart’s normal rhythm immediately and automatically,” he added.

According to the researchers, this could represent a great improvement on the current way of stopping atrial fibrillation.

The heart attack brings about activation of certain genes which stay as a permanent memory in genes. Pixabay

Atrial fibrillation is the most common heart rhythm disorder in clinical practice. The current treatment, known as cardioversion, is based on administering an electric shock to the heart, which has to be done in the hospital under general anaesthesia because of pain.

For many patients, this is the only treatment to immediately stop atrial fibrillation because drugs or an operation are ineffective.

“The bioelectronic defibrillator can stop atrial fibrillation without an electrical shock. In this way, the heart can be reset in a fully automated manner and at any time,” Pijnappels said, in the paper reported in the journal Science Translational Medicine.

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“We anticipate that this treatment for atrial fibrillation could improve both the patient’s quality of life and their prognosis,” he added.

However, considerable research is still needed before a treatment can be arrived at that is suitable for human patients, the researchers noted. (IANS)

Next Story

Scientists Develop “Special Molecular Switch” for Gene Therapy

In a study, the researchers demonstrated the power of their new switching technique by incorporating it into a gene therapy that produces the hormone erythropoietin

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However, gene therapies have been viewed as inherently risky because once they are delivered to a patient's cells, they cannot be switched off or modulated. Pixabay

Paving the way for solving a major safety issue associated with gene therapies, scientists have developed a special molecular switch that could be embedded into such therapies to allow doctors to control dosing.

The feat, published in the journal Nature Biotechnology, offers gene therapy designers what may be the first viable technique for adjusting the activity levels of their therapeutic genes.

The lack of such a basic safety feature has helped limit the development of gene therapy, which otherwise holds promise for addressing genetically based conditions.

“I think that our approach offers the only practical way at present to regulate the dose of a gene therapy in an animal or a human,” said lead researcher Michael Farzan from the Scripps Research Institute in Jupiter in Florida, US.

Gene therapies work by inserting copies of a therapeutic gene into the cells of a patient, if, for example, the patient was born without functional copies of the needed gene.

The strategy has long been seen as having enormous potential to cure diseases caused by defective genes. It could also enable the steady, long-term delivery to patients of therapeutic molecules that are impractical to deliver in pills or injections because they don’t survive for long in the body.

Gene
Paving the way for solving a major safety issue associated with gene therapies, scientists have developed a special molecular switch that could be embedded into such therapies to allow doctors to control dosing. Pixabay

However, gene therapies have been viewed as inherently risky because once they are delivered to a patient’s cells, they cannot be switched off or modulated. As a result, only a handful of gene therapies have been approved to date.

In this study, the researchers demonstrated the power of their new switching technique by incorporating it into a gene therapy that produces the hormone erythropoietin, used as a treatment for anaemia.

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They showed that they could suppress expression of its gene to very low levels with a special embedded molecule, and could then increase the gene’s expression, over a wide dynamic range, using injected control molecules called morpholinos that the US Food and Drug Administration has found to be safe for other applications. (IANS)