Behind the Scenes of Gene Therapy by Kelly Hongkham


“Gene therapy” – what is the significance of these two words?

For many, it spells out hope for a cure, for a chance at a healthier life, but at the Nolta lab, it is that and more. Here, the creation of gene therapies is a major basis of all the hard work and research that the Nolta team has dedicated their time to for over 30 years.

The ultimate goal is to treat currently incurable diseases and disorders through the manipulation of genetic material.

Simple enough, right?

Of course not.

Creating a successful gene therapy requires much more than targeting a defective gene and inserting a corrected one. In fact, just thinking about the multitude of research that is done every day in order to get just a little bit closer to a solution is simply amazing.

So, how does it work?

Over the past 8 weeks, I was given the incredible opportunity to peek into this world of research through the CIRM SPARK program, an internship which allows high school students to gain valuable experience working alongside professionals in a state-of-the-art research facility.

In all honesty, I was a bit overwhelmed. From day one, the other interns and I were taught all the ins and outs of working in the lab: what to do, what not to do, where to find this, where to store that, who to contact if something goes wrong, and, of course, the number one rule – ALWAYS wear your personal protective equipment. At first, I was worried because, knowing me, I would be the one to forget something and make a mistake. However, I quickly came to understand how friendly and supportive the Nolta team is and in no time, interning there became the perfect balance between hard work and fun.

At the Nolta lab, I had the honor of working in the Karyotyping Core alongside my amazing mentor, Catherine Nacey. From her, I learned the techniques for chromosomes analysis. Karyotyping is just one of the many molecular assessments important for quality control.

Hongkham, Kelly hood

For me, the most challenging part of the karyotyping protocol was learning how to identify each of the 23 pairs of human chromosomes. Now, even though you might think that they could be identified just by length, it definitely isn’t that easy. Believe me. However, fortunately, my mentor was very patient with me and within 2 weeks, I went from “Well… that’s definitely a chromosome” to “That’s an 11, I mean look at that wide heterochromatic region. And those dissipating bands on the q arm, come on it’s so obvious”.

Okay. Maybe I’m exaggerating just a little, but my mentor definitely has that level of confidence. Even now, I am still amazed by how rapidly she is able to identify chromosomes on even the worst-quality metaphases.

So, what happens from there?

After quality control, what’s the next step?

I could tell you briefly about how other research works, but it would not be as in depth as what I could tell you about karyotyping from harvesting the cells, to making the slides, to the final report. Even with all that I have learned this summer about gene therapy research, there is still so much that I do not know, and after getting a taste of what it is like, I want to know more. I have been inspired by all these passionate researchers and I look forward to being a part of the exciting gene therapy breakthroughs that will occur during my lifetime.




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