How Human Longevity Advances Can Help Kids With This Rare Genetic Disorder
As a society, we try our hardest to stay young. Both mentally and physically, we do our best to stay in shape and live healthy lives in order to maximize our time here on earth. Inevitably, we all grow up, but our work to stay young pays off to an extent because we can spend our lives in good health. But, what if I told you that there are some kids who have no control over how fast they age? Right now, children facing this rare condition live a fifth of the average lifespan, yet die with the bodies of an average seventy year old.
Today, we’re going to discuss a genetic condition called Hutchinson-Gilford Progeria Syndrome, also known as progeria. Then, we’ll dive into what longevity can do to help kids who face this condition.
What is Progeria and How Does it Affect Kids?
The world is filled with all different types of people. People of different races, ethnicities, sizes, appearances, cultures, and nations. And each individual person is unique. Some people face challenges that make them a bit more visibly unique than others. To put this into perspective, let’s take a look at a comparison:
Pictured above is a child. Just an average, healthy, happy kid.
Again, pictured above is a child. Like the first kid, he’s happy and adorable. But, there’s something different about him. He has progeria.
And I’m sure you’re wondering: what is progeria?
progeria is a rare genetic condition, suffered by one in four million babies, causing them to age at an accelerated rate. At just two years old, they appear to be more than ten times their age. They develop alopecia, which causes baldness. And their internal organs resemble that of an elderly person. As a result, they are at an extremely high risk for diseases and conditions that are usually common to people over sixty. Many of these kids have atherosclerosis, which occurrs when the arteries are clogged because of plaque buildup. So, these kids often pass away from heart disease. And on average, children with progeria only live to be thirteen.
Kids with progeria develop different physical and internal characteristics from average children. These differences can be understood through the diagram below:
So, What Causes Progeria?
Progeria is caused by a mutation in the LMNA gene, which codes for the lamin A protein. The lamin A protein is the scaffolding that holds the nucleus of a cell together. Researchers believe that the defective lamin A protein makes the nucleus unstable, causing cellular instability. This leads to the production of an abnormal protein called progerin that interferes with cell division. And this interference causes cells to break down more easily and rapidly without being replaced, leading to a breakdown of the body’s organ systems. This causes the process of premature aging.
What’s the Cure?
Well, unfortunately there isn’t one. BUT, that can change and there is hope for these kids.
Ordinarily, when a gene mutation causes a disease, scientists would try to correct the gene. However, conventional gene therapy that involves adding genes can’t help with progeria because the issue is the mutant progerin protein.
Rather than using gene therapy, another method of cell repair would be to implement stem cell therapy. Scientists have made significant strides in the field of human longevity and using stem cells to rejuviante people to live healthier and longer lives. This same technology will allow them to further contribute to finding a cure for progeria.
IPS Cells:
Our bodies are made up of cells, all of which have set specific functions. Once a cell is assigned a type at a baby’s birth, it functions as it should, and cannot change to a different type of cell with a different function. But, stem cells are special because can become any type of cell, functioning as that cell would, and can also almost endlessly divide and form new cells to build and repair tissues and organs in the body. By doing so, their job is to act as regenerators for other types of cells. However, stem cells age as we do. The older we get, the effectiveness and amount of stem cells we have decrease exponentially. So understandably, the health of stem cells in kids with progeria isn’t that great.
Scientists have discovered that genetic reprogramming can be used to program adult stem cells back to an embryonic state. Embryonic stem cells are the most versatile because a baby’s cells are still learning what to do when they are conceived. And, these cells are pluripotent, meaning that they can grow into any type of tissue in the body. But, adult stem cells are less versatile because although they can become many different types of cells, they can’t become every type. This is where induced pluripotent stem cells come in. These cells are reprogrammed to act in the same way as young stem cells. They are injected into patients via an IV and can significantly repair damage in the body.
This reprogramming process can be slightly altered to help patients fighting progeria and potentially lengthen their lifespans.
So, a regular IPS reprogramming process can reprogram cells. The problem with this is that if progeria stem cells are reprogrammed, the stem cells themselves willl still have progeria. When they turn into other types of cells, those cells once again produce the bad lamin A and once again cause pre-mature aging. Instead, a different approach is needed. Gene therapy can shut down the expression of progerin in progeria patients’ stem cells. By using gene therapy, all cellular defects can be ereased from the progeria patients stem cells to produce rejuvenated pluripotent cells that function as healthy cells.
What would the process result in?
Essentially, by first using gene therapy to alter the make up of the stem cells, and then genetically reprograming the stem cells back to an embryotic state and injecting them into the progeria patients, kids with progeria will be able to reguvinate their bodies.
In the End:
Ideally, these kids will be able to live disease-free, long lives. However, as we all know, science is a long and complicated process. And, my hypothesis is not 100% accurate. It will take time and much more research to perfect this idea. And with that being said, maybe one day this type of stem cell therapy can be applied to everyday people in their 60s and 70s who face similar health problems to kids with progeria. Ultimately, we will be able to bring the idea of human lingevity to life, one step at a time.
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