Tag Archives: Gene therapy

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.


New gene therapy for heart damage goes to clinical trial

English: Coronary circulation, with coronary a...
English: Coronary circulation, with coronary arteries labeled in red text and other landmarks in blue text. This vector graphics image was originally created with Adobe Illustrator, and modified with Inkscape. 32px|alt=W3C|link=http://validator.w3.org/? The source code of this SVG is valid. Category:Valid SVG (Photo credit: Wikipedia)

Researchers at Saint Louis University have had such promising results from a small (40 patients) earlier study using gene therapy for repairing heart failure damage that they’re now instituting a clinical trial.  The trial will use a genetically targeted regulatory protein replacement therapy to see how well it can repair damage in 200 patients who’ve either had a prior heart attack or have cardiomyopathy or advanced heart failure.

The therapy involves infusing a gene – a type of regulatory protein called SERCA-2a – down the coronary arteries that may help heart cells utilize calcium better, which is similar to an engine getting better gas mileage. This outpatient procedure is performed in a cardiac catheterization lab and aims to increase the level of SERCA-2a, which is reduced in the individual heart muscle cells in patients with heart failure and can thereby change the way those cells function.

Worldwide 52 sites will participate in the trial, 33 in the U.S.  To participate in the trial, please contact the Cardiology Research Office at Saint Louis University at 314-577-8876. Read the original article here.

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Trials underway using stem cell and gene therapy to treat heart damage

Heart Failure causes more damage than you think
Heart Failure causes more damage than you think (Photo credit: Novartis AG)

Both Canada and the UK have recently begun large-scale human trials to test how well new approaches using stem cells and gene therapy will work for treating damage in human hearts.

The idea of the Canadian study is that a patient’s own stem cells are the most direct and effective way to repair damage and rebuild function in the heart. But because the stem cells from a damaged heart are not working up to normal capacity, scientists tested and found that adding extra copies of a gene that “stimulates blood vessel growth and improves tissue healing, known as endothelial nitric oxide synthase,” improves that function.

In other words, the gene stimulates the patient’s stem cells to reproduce more quickly and do their magic to help the heart heal itself. The Canadian trial is for post-heart-attack patients.The UK trial will be using a carrier virus to insert a gene into heart failure patients to help their hearts pump better.

Nothing but good news here – except that it will be two and three years before results are in. Stay tuned.

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Stem cells and gene therapy restore hearing, vision, smell in animals

English: Diagram to show how embryonic stem ce...
English: Diagram to show how embryonic stem cells are differentiated (Photo credit: Wikipedia)

A researcher spent ten years and finally succeeded in getting human stem cells to grow into two distinct types of cells—auditory neurons and inner-ear hair cells. And now he’s used the appropriate neuron-type cells to re-connect the inner ear to the brain. In other words, to restore nearly 50% of hearing in gerbils (whose inner-ear hairs had remained undamaged).

It’s very niche research, but it demonstrates that restoring hearing is definitely possible to some degree. Plus other research shows it’s possible to restore hearing to mice born deaf and yet other gene therapy research showing you can restore function to hair, eye and heart cells and smell in mice.

So much of historical medical research has focused on devising invasive, even barbaric methods of arresting sickness. We refer to it always as “fighting” disease, killing cells, conjuring up images of swords, bullets and bombs. As we continue to plumb the magical powers of stem cell and gene therapies, it’s encouraging to think of the balance now slowly tipping more and more toward non-invasive ways of restoring, gently giving back, quality of life to those who suffer.

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Gene therapy shows big promise for advanced heart failure

DNA vaccine and Gene therapy techniques are si...
Image via Wikipedia

It’s only a small group of patients, but the results are dramatic. A new gene therapy for advanced heart failure looks really promising.

People can live a long time with heart failure if it’s kept under control. But if it gets to where they can’t keep the water from accumulating beyond safe levels in the body’s tissues, patients begin to feel like hell and experience more frequent cardiac events that put them in the hospital.

Researchers conducted a Phase II trial at Mount Sinai School of Medicine with a gene therapy developed there and found it stabilized or improved cardiac function in people with severe heart failure. The patients who were given a high dose of the therapy, called SERCA2a, benefited clinically (which means they felt better or lived longer) and had significantly fewer cardiovascular hospitalizations. The study appears online in the June 27 issue of the American Heart Association journal Circulation.

Simply put, the SERCA2a therapy consists of delivering an inactive virus that carries medication into cardiac cells. It then stimulates the heart cells to produce an enzyme that helps the heart pump more effectively in people with advanced heart failure.

Quality of life is often just as important as longevity. If you can feel okay and not have to go to the hospital every other week or month, it’s a lot easier to live your life more fully. Advanced heart failure is tough—it’s always exciting to see that science continues to find ways to use the tools of nature to help in relatively non-invasive ways.

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