Scientists Develop Injectable Gene Therapy To Cure Genetic Condition

Health Blog

Some people have been blessed with good health. Then, there are those who have worked hard for it. As the old adage goes, “Health is wealth.” Simply put, health will always make your life easier. Unfortunately, this is difficult for some to achieve.

There are many out there that have been diagnosed with a genetic condition. Finding a cure for this isn’t always possible. Researchers have been looking for a way to address this issue, and hope may not be in the horizon for them.

What happens to those who suffer from a genetic heart condition? To start with, a crack team of scientists are ready and armed with a sizeable research grant. Their ultimate goal is to develop an injectable gene therapy that is able to address one of the most difficult diseases known to modern science.

Genetic cardiomyopathies is a part of a group of diseases that are genetically passed on to offspring. This is at a 50/50 chance. When the child is born with a faulty genes, this may bring about heart failure that affects the young adults as they go through their prime years. In the UK alone, this happens at a rate of 12 per day for those under 35 years old.

A team of global experts from the UK, US, and Singapore work at CureHeart and they have recently been awarded a whopping £30 million grant from the British Heart Foundation. This will go to the development of a treatment to reprogram the genetics that brings about muscular heart disease to a certain number of people in the population.

The grant was given to the group based on preliminary work in animal studies. The results of these studies have shown a single-shot treatment based on CRISPR technology called “base and prime editing” can address and even cure various genetic ailments of the heart. This is extremely important because the health issue affects 1 in every 250 people worldwide.

Professor Gavin Screaton, the Head of the Medical Sciences Division, University of Oxford said, “This is an extraordinary opportunity for the CureHeart team to make a difference in the lives of so many patients across the world. We have a long history of multidisciplinary working here in the Medical Sciences Division, and pride ourselves in the many successes our scientists have had in translating their cutting-edge lab research into real life treatments. This work by Professor Watkins and the international CureHeart team exemplifies this ethic at its best and we are truly excited to see their progress.”

The shot of the injectable is for correcting genes that produce abnormal proteins in the pumping machinery found at the heart. The gene that causes myopathy could be re-written or switched off for good. That is, depending on what type it is.

As for where genes are not producing enough proteins that are able to support a healthy heart that is functioning, the injectable will be able to correct the issue by two ways: correcting the faulty copy or reinforcing it.

“This is our once-in-generation opportunity to relieve families of the constant worry of sudden death, heart failure and potential need for a heart transplant,” said Professor Hugh Watkins. He’s from the University of Oxford. He’s also the lead investigator in the aforementioned project that’s set to begin.

“After 30 years of research, we have discovered many of the genes and specific genetic faults responsible for different cardiomyopathies, and how they work. We believe that we will have a gene therapy ready to start testing in clinical trials in the next five years.”

“The £30 million from the BHF’s Big Beat Challenge will give us the platform to turbo-charge our progress in finding a cure so the next generation of children diagnosed with genetic cardiomyopathies can live long, happy and productive lives,” he added.

As for Dr. Charmaine Griffiths who is the Chief Executive of the British Heart Foundation, she has a lot of hope for this project. She said, “With the public’s support, the aim of the Big Beat Challenge was to move past incremental progress and make a giant leap in an important area of heart patient care. Creating the world’s first genetic cure for a heart disease would undoubtedly do this and has the potential to stop families losing loved ones without a moment’s notice to these cruel diseases. However, we need the continued backing of our supporters to turn science like this into a reality for the millions of people around the world living with heart disease.”