08:43am Saturday 07 December 2019

Medical Miracles: What is Gene Therapy and Why You Should Care

The pace of progress being made in new medical treatments and techniques is astounding and gene therapy is a good example of how far we have come in offering patients viable options and alternatives to improve their health.

Gene therapy is considered a real game-changer by a growing number in the medical profession as it creates the opportunity to treat a patient without the need to use conventional drugs or surgery.

Here is a look at what gene therapy is and how it can make such a meaningful difference when it comes to treatment options, including an overview of where we are at with testing, and some examples of the research and results that have been reported so far.

Gene therapy explained

In general terms, a good way of describing gene therapy is to currently call it an experimental technique that involves using genes as a way of treating or preventing disease occurring in the first place.

We are still discovering the power and the possibilities of gene therapy so the technique is by no means the finished article at this point, but what we know so far makes it a very exciting medical development that could really change the way we are treated for certain conditions and hereditary diseases.

This is a collaborative drug discovery scenario that is evolving as researchers test several different approaches to gene therapy.

There are several gene therapy techniques that are being pursued in particular, with others being trialed as we continue on this medical voyage of discovery.

One technique involves replacing a mutated gene that causes disease with a healthy copy of the gene instead. Another method being used is to find a way of deactivating a mutated gene when it has been identified as functioning improperly.

Also, scientists have been working on a technique that involves introducing a new gene into your body as a way of helping you to combat a specific disease.

It is fair to say that amidst the great excitement surrounding the huge possibilities offered by gene therapy treatments as a way of fighting a variety of diseases, certain cancers, and a number of viral infections, the technique is still in the testing stage to ensure that it is safe and effective in equal measure.

This means that the current situation with gene therapy is that it is only being tested for its effectiveness in treating diseases with no other known cures, before being rolled out to other areas of health.

Using gene therapy to restore hearing

Although gene therapy has not become a mainstream option just yet there is a lot of research work going on to bring that watershed moment closer every day, with some experiments demonstrating the huge potential of the treatment.

One example of this surrounds the use of gene therapy to restore hearing in deaf mice.

A team of scientists based at Boston Children’s Hospital have reported being able to restore rudimentary hearing capabilities into genetically deaf mice. The research has been progressing at a decent pace and using an improved gene therapy vector developed at Massachusetts Eye and Ear, they have managed to restore a high level of hearing that extends down to 25 decibels, which is barely a whisper.

It is incredible to think that someone who is deaf could potentially have their hearing restored to such an extent that they could even pick up what someone was whispering.

Combating macular degeneration

Another interesting development has been the use of gene therapy to prevent blindness.

Age-related macular degeneration (AMD) affects a point at the back of your eye known as the macula, which is on the retina. When AMD impacts your vision it becomes blurred and distorted, with black spots appearing in the center of your eyesight.

At least ten percent of the population aged over 65 are affected in some way by AMD, so the scientists who are performing gene surgery on mice in the layer of tissue supporting the retina, will no doubt be encouraged that if the trials continue to yield positive results there is a going to be a gene therapy solution that could be offered to resolve AMD issues in the near future.

These are just a couple of example of the continuing work that is going on to find ways to use gene therapy as a viable way to combat disease and even prevent it from occurring at all when it becomes possible to replace or deactivate faulty genes.

Alice Noble works in the medical industry and has articles published on a growing number of health and medical themed blogs.

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