A Brief History of Gene Therapy for Students in Pharmaceutical Training


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The preconceived idea of pharmaceutical products usually revolves around pills, creams, liquids, or powders. Treatment for medical conditions is, however, much more broad, and gene therapy is one such option that is receiving more and more attention. Despite being around for many decades, experts have struggled to overcome the side effects of this revolutionary treatment. The benefits could have far-reaching consequences for patients with cancer, cystic fibrosis, and many more conditions for which there are currently limited treatment options or no cures.

It’s an exciting alternative to existing drugs and surgery, so pharmaceutical professionals are keeping a close eye on the latest developments with this form of treatment. To understand its benefits and risks, it’s necessary to reflect on the history of gene therapy, so let’s take a closer look at its progression through the decades.

The Science Behind Gene Therapy Techniques

As the name would suggest, this technique uses healthy DNA to try and replace troublesome genes in the body. Vectors must be used to transport the new gene into the cell’s genome. These vectors are adapted viruses which can offload the new gene when it comes into contact with the cell.

There are a few different versions of gene therapy which are each suited to particular medical tasks. Gene augmentation therapy adds a healthy gene into the malfunctioning gene in order to help the existing version function normally. This is seen as a potentially important step in the treatment of cystic fibrosis, a condition which attracts lots of research from graduates of pharmaceutical training.

Gene inhibition therapy is used to block the activity of a rogue gene in a cell, while there are also options to kill specific diseased cells. These techniques are better suited to treating conditions such as cancer, where groups of cells need to be neutralized or destroyed.

Rogue cancer cells could be overcome through gene therapy

Rogue cancer cells could be overcome through gene therapy

The Rise and Fall of Gene Therapy as a Treatment Option in Its Early Years

The initial idea behind gene therapy was first mooted in 1972 when researchers suggested it as a treatment for genetic diseases. It was still much too soon to carry out such an experiment, and it was only in 1990 that a four-year-old girl with an immune system deficiency was first treated with gene therapy in the USA. There was a guarded welcome to the results, but experimentation took a turn for the worst at the end of the century.

The death of Jesse Gelsinger is an infamous moment in the history of gene therapy. The 18-year-old American died four days after starting treatment and it’s believed that he responded badly to the vector carrying the new gene. The FDA held back on further experimentation in the aftermath of the tragedy. The credibility of gene therapy was damaged further in the early 2000s when some French children were diagnosed with leukemia after clinical trials.

Students in Pharmaceutical Training Are Noting the Resurgence of Gene Therapy

Despite those significant setbacks, research has continued into the potential benefits of gene therapy. China approved the technique for head and neck cancer treatment in 2003, and it was first used on a genetic defect four years later.

Those completing a pharmaceutical quality assurance diploma recognize that there is plenty of momentum behind the support for increased use of gene therapy. The European Union backed agency, EMA, became the first western authority to approve gene therapy treatment in 2012, namely Glybera for a rare blood disorder. Canadian researchers have also discovered a treatment which offers exciting benefits for sufferers of diabetes. There is still some skepticism surrounding this type of treatment, but its potential advantages mean that it will continue to attract interest from the medical sector well into the future.

Gene therapy can have benefits for those with cystic fibrosis

Gene therapy can have benefits for those with cystic fibrosis

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