Is This the Beginning of Gene-Editing Medicine?

Does the smiling face of Layla Richards mark a new era in genetic medicine that could change all our lives?

The story of Layla Richards may be just the beginning of a new chapter in medicine.

Layla Richards developed CLL as an infant and the day before her first birthday, Layla's parents were told she was going to die. All treatments for her leukemia had failed.

An experimental treatment that had only been tried on mice was her only hope. Her family, her doctors and a biotechnology company decided they had to try it.

The biotechnology company genetically engineered some cells that were designed to kill Layla’s cancer.
Today, because of this experimental treatment, Layla has no trace of leukemia.

We could be seeing the start of a revolution in medicine. The same procedure that worked in Layla, could be used to treat, not only other cancers, but inherited diseases.

Genetic engineering is a whole new field in which missing DNA be inserted and defective DNA can be corrected. This is what doctor’s did for Layla.

This revolution may have started around the turn of the millennium when scientists were saying that gene therapy was going to transform the world. While it has taken 15 years for gene therapy technology to develop, the experimental treatment that saved Layla’s life may be just the first miracle of this new technology.

As with all advances in science, caution is the word for the day. Gene-based therapy involves changing our DNA. These are the instructions for building and running every part of our body.

Early attempts to use this technology ultimately resulted in patients developing leukemia and further attempts were abandoned.

DNA was being inserted, in such a way, that it disrupted the natural functioning of some cells and they became cancerous.

Since then the process has become much more precise. The viruses being used can precisely place DNA into safer sites in the genome. This new precision has been described as the difference between using an ax and a laser. The viruses used act as a guide that finds its way to specific sites in our DNA and a pair of molecular scissors that can edit the DNA.

Will doctors be able to harness our DNA for new treatments?

A similar technique is already being tested in HIV-positive patients. The aim is to take the patient's cells out of the body, give them HIV protection, and then put them back in.

The doctor involved in treating Layla's leukemia, told the BBC: "The technology is moving very fast, the ability to target very specific regions of the genome has suddenly become much more efficient.

"The technology itself has got enormous potential to correct other conditions where cells are engineered and given back to patients or to provide new properties to cells that allow them to be used in a way we can only imagine at the moment."
Re-arming the immune system to target cancer and a wide range of inherited disorders is in the sights of doctors.

It will be easiest for them to use cells that can be taken out of the body, modified and reinserted rather than trying to edit them still in the body.

So diseases of the blood or immune system - such as MPNs or sickle cell anemia - will be easier targets than kidney or heart defects.

Doctors are predicting an "explosion" in the use of such genetic engineering in the next 10 years.

After the overhyped false dawn fifteen years ago, gene-editing is now, it seems, about to arrive.

Author: Crowley, William

Bill Crowley has worked with blood cancer patients, family and friends for over 15 years. As the Director of Development, he helps supporters appreciate how their gifts impact MPN research.

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