By: Ira Lako, MSCR
Senior Associate Scientist II, Voyager Therapeutics
While it is no secret gene therapy is making a comeback in the science community, the novel mechanisms of gene editing and its associated scientific jargons can get confusing quickly, even for an experienced scientist. Inherently, this begs the question, what is gene therapy and how does this even work? And perhaps, more importantly, why should we care?
Now, before we fast forward to the end, we must first look into the origin of this story. Our story begins where the merging of two different gene sets come together to create one human life. These genes are the basis of everything that make up a person, from appearance like eye color to internal functions like metabolism. If humans were computers, our genes are the codes we have pre-installed. Unfortunately, while we may think nature is perfect, she too has her flaws. Sometimes genes do not get passed down correctly and that creates a mutation in the set. It is to be noted some mutations are passed down from generation to generation, some are made for the first time, and not all mutations are inherently bad! Heterochromia, or the condition where someone has two different eye colors, is a great example of this. It is both a mutation and a trait, that apart from the physical difference, poses no threat to the human carrying the mutation. But what happens when a mutation conversely has a negative effect? An example of this is Huntington’s Disease; a faulty gene set causes premature brain cell death that in turn, severely shortens one’s life expectancy. This is where our main character gene therapy enters the chat\!
Gene therapy targets the faulty genes within the cells. A “correct” set of genes that would produce the functional output desired is then supplied to the patient through a delivery vehicle called a vector. While it does not remove the pre-existing “faulty” gene set, the addition of the “functional” gene set allows for the correct code to be sent out to the body to produce the correct proteins needed and therefore correcting the disease set by the mutation in the first place. This gives not only hope, but another chance at life for some patients. Personally, where innovation meets humanity is the crux of science. We may not have answers for everything, however, this is a start to leaving the world better and more capable than we found it!
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