In this episode, we explore some of the major scientific findings – like discovering DNA – that set the stage for the development of gene therapy and its groundbreaking potential when it comes to the treatment of genetic diseases. 

The very idea of gene therapy wouldn’t be imaginable had two pairs of pioneering scientists not bonded decades earlier. In 1951, a young chemist named Rosalind Franklin and her colleague Maurice Wilkins at King’s College in London were using X-ray crystallography to try and perceive the properties of a theoretical molecule known as deoxyribonucleic acid.

At the time, many scientists believed that all the genetic information about living organisms was contained in a molecule called DNA. But no one had figured out exactly what it was, or what it looked like.

After attending a presentation by Franklin, James Watson – who was also studying the topic – connected with Francis Crick. Crick had been studying the concept of base pairs – the idea that nucleic acid is composed of chemical bonds between not one but two sets of molecules, each supporting the other, much like the two sides of a ladder support the rungs in between. Excited by their shared passion, Crick and Watson in Cambridge began to build models of possible DNA structures, trying to figure out just how all the pieces fit. 

Eventually, Franklin, Wilkins, Watson and Crick’s efforts joined, and DNA was discovered. Then, in the 1970s, DNA was successfully transferred from one life form to another.

Less than 50 years after Crick, Franklin, Wilkins, and Watson first showed us what this molecule looks like, genetic engineering gave us the ability to reprogram it when it isn’t working. 

Scientists and doctors began to dream big: could this technology eventually cure all genetic diseases?

There was still work to be done. But, like a tiny plasmid loaded up with recombinant DNA, we were on our way.

For more education on gene therapy, visit www.genetherapynetwork.com.