This week’s roundup highlights how we keep finding new and exciting ways stem cells could help change how we treat disease.
Our Cool Stem Cell Image of the Week comes from our first story, about unlocking some of the secrets of Huntington’s disease. It comes from the Laboratory of Stem Cell Biology and Molecular Embryology at The Rockefeller University
A new approach to studying and developing therapies for Huntington’s disease
Researchers at Rockefeller University report new findings that may upend the way scientists study and ultimately develop therapies for Huntington’s disease. Huntington’s is a devastating, inherited neurodegenerative disorder that has no cure. The team focused on human biology because human brains are more complex than mice. So, they used CRISPR gene-editing technology in human embryonic stem cells to introduce the genetic mutations that cause HD.
Although symptoms typically do not appear until adulthood, the researchers were surprised to find that in their human cell-based model of HD, nerve-cell abnormalities occur at the earliest stages of brain development. These results suggest that HD therapies should focus on much earlier interventions.
The researchers observed another unexpected twist: cells lacking Huntingtin, the gene responsible for HD, are very similar to cells from HD patients. This suggests that too little Huntingtin may be causing the disease. Up until now, the prevailing idea has been that Huntington’s symptoms are caused by the toxicity of too much mutant Huntingtin activity.
We’ll certainly keep an eye on how further studies using this new model affect our understanding of HD and therapy development.
This study was published in Development and picked up by ScienceDaily.
How you can double your DNA
As you can imagine, we get lots of questions about stem cell research here at CIRM. Last week we got an email asking if a stem cell transplant could alter your DNA. The answer is, under certain circumstances, yes, it could.
A fascinating Herald Review article explains how this can happen. In a bone marrow transplant, doctors kill bad blood stem cells and replace them with healthy ones from a donor. As those cells multiply and create a new blood supply, they also carry the donor’s DNA.
But that’s not the only way that people may end up with dual DNA. The really fascinating part of the article is how this can create all sorts of legal and criminal problems.
One researcher’s efforts to reverse aging
“Stem cells are the Gary Oldman of cell types.” As a fan of Gary Oldman (terrific as Winston Churchill in the movie “Darkest Hour”), that one line made me want to read on in a profile of Stanford University researcher Vittorio Sebastiano.
Sebastiano’s goal is, to say the least, rather ambitious. He wants to reverse aging in people. He believes that if you can induce a person’s stem cells to revert to a younger state, without changing their function, you can effectively turn back the clock.
Sebastiano says if you want to achieve big things, you have to think big:
“Yes, the ambition is huge, the potential applications could be dramatic, but that doesn’t mean that we are going to become immortal in some problematic way. After all, one way or the other, we have to die. We will just understand aging in a better way, and develop better drugs, and keep people happier and healthier for a few more years.”
The profile is in the journal Nautilus.