Category Archives: gene therapy

Marker in cells mutated back to stem-cell-state may be able to predict cancer

English: Pathway of stem cell differentiation
English: Pathway of stem cell differentiation (Photo credit: Wikipedia)

News of a marker that may serve as a way to predict which cells will become cancer comes out of a recent study by researchers at Boston Children’s Hospital. Scientists were able to isolate a substance that appeared in a cell that had reverted to its stem-cell-state and then became cancerous in zebrafish that subsequently developed melanoma.

“The spark for that change was in the crestin gene, which should only be active in embryonic tissue but became inappropriately activated again, resulting in melanoma,” according to the article online at One of the authors of the study said “the beginning of cancer occurs after activation of an oncogene or loss of a tumor suppressor, and involves a change that takes a single cell back to a stem cell state.”

Now a fish is not a human being, so this information is a long way from having practical application in your doctor’s office. But the principle behind this discovery will guide further research and additional hoped-for developments.

Stem cells made to produce new neurons

English: Complete neuron cell diagram. Neurons...
English: Complete neuron cell diagram. Neurons (also known as neurones and nerve cells) are electrically excitable cells in the nervous system that process and transmit information. In vertebrate animals, neurons are the core components of the brain, spinal cord and peripheral nerves. (Photo credit: Wikipedia)

Our bodies are born with X number of neurons, which are the parts of the nervous system that carry chemical and electrical messages between the brain and other body parts. Up until now, no one has found neurons reproducing like regular cells do.

Now scientists have found a way to manipulate an RNA molecule called Pnky in such a way that neural stem cells will actually produce new neurons. It’s a complex process that takes a lot of explaining, so read more at And while you’re at it, read some clear layperson’s explanations about the many functions neurons perform in our bodies and brains on

They’ve only done this in mice so far. But it’s clear this discovery constitutes a breakthrough in understanding a whole set of long, non-coding RNA molecules that researchers have heretofore assumed were just there–with no known function. One scientist called these the “dark matter” of the human genome, meaning there’s a lot more of this stuff than there is of the DNA we’ve already explored and named.

Another whole new world of knowledge is just beginning to crack open.

Stem cells repair older muscle, gene changes cells to restore heart rate

Mouse embryonic stem cells. More lab photos
Mouse embryonic stem cells. More lab photos (Photo credit: Wikipedia)

Can’t keep up with all the exciting research going on with stem cells and gene therapy. I imagine many of the researchers might feel a little like a kid who just discovered clay – and found you can do almost anything with it.

First up is a project in which researchers identified why older creatures can take so long to recover from muscle injuries. They found that muscle stem cells in mice were not dividing and renewing themselves the way younger stem cells do.

So they found a way to reinvigorate the murine stem cells by blocking a substance known as p38 MAP kinase that appears to be critical in slowing stem cell division. The growing medium, soft hydrogel, is critical to the newly invigorated cells maintaining their “stemness.” They then injected the revunated stem cells into damaged muscles of elderly (two years old – about the same as 80 in humans) mice and found the muscles began substantially repairing themselves.

Next up, pigs who were suffering from heart block (their heart rate was too slow) were injected with a human gene into a tiny area of the pumping heart that allowed researchers to reprogram “heart muscle cells into a type of cell that emits electrical impulses to drive the beating heart.” The pigs’ heart rates were restored to normal for two weeks.

The hope is that these “biological pacemaker” cells will one day replace the devices we now insert into bodies that must have batteries replaced every 8 to 10 years. Read more about pacemaker gene therapy here.