Tag Archive for 'Spinal muscular atrophy'

USA – Stem Cells, Possible to Reproduce Diseases in Laboratory

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An experiment successfully performed by researchers at the University of Wisconsin-Madison and the University of Missouri in Colombia, described in Nature magazine shows that cells removed from a patient’s skin and transformed into cells similar to embryonic stem cells have become a laboratory model for diseases and can be observed in real time and studied to find new cures. The researchers recreated spinal muscular atrophy (SMA) pluripotent stem cells removed from the skin of a child affected by the neurodegenerative genetic disease. In the laboratory, the cells behaved exactly as they do in a human being, giving the researchers an opportunity to observe the progression of the disease. This is an entirely new opportunity, made possible by a discovery by a Japanese research group, which less than a year ago was able to reprogram adult embryonic-like stem cells.

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Stem Cells: From skin cells to motor neurons

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A team of Harvard stem cell researchers has succeeded in reprogramming adult mouse skin cells directly into the type of motor neurons damaged in amyotrophic lateral sclerosis (ALS), best known as Lou Gehrig’s disease, and spinal muscular atrophy (SMA). These new cells, which researchers are calling induced motor neurons (iMNs), can be used to study the development of the paralyzing diseases and to develop treatments for them.

Producing motor neurons this way is much less labor intensive than having to go through the process of creating induced pluripotent stem cells (iPSC, iPS cells), and is so much faster than the iPS method that it potentially could reduce by a year the time it eventually takes to produce treatments for ALS and SMA, said Kevin Eggan, leader of the Harvard team.

Importantly, the direct reprograming does not involve the use of any factors known to trigger cancer or any other disease states, and the factors in fact make the fibroblasts, the connective tissue cells that make and secrete collagen proteins, stop dividing.

The work by Eggan, a member of the Harvard Stem Cell Institute principal faculty and an associate professor in Harvard’s Department of Stem Cell and Regenerative Biology (SCRB), and his colleagues builds on and advances work by SCRB co-chair and Professor Doug Melton, who pioneered direct cellular reprogramming, and Marius Wernig of Stanford, who used direct reprogramming to produce generalized neurons.

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Stem Cell Research Using Adult Stem Cells for Spinal Muscular Atrophy

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A young 16 year old boy with Type 2 Spinal Muscular Atrophy has returned home with noticeable improvements after receiving Adult stem cell research in China. Kyle Knopes, from Janesville, Wisconsin says his quality of life has improved tremendously after returning from China where he received adult stem cells taken from cord blood.

What is Spinal Muscular Atrophy?

Spinal Muscular Atrophy is a hereditary disease in which the person is missing a gene which is responsible for the production of the protein for motor neurons. Without this protein, the motor neuron in the spinal cord wither and die.

Before the Stem Cell Treatment in China

  • Couldn’t open his fist
  • Couldn’t roll over on his side by himself
  • All muscles were very weak, could only hold empty cups

After the Stem Cell Therapy Using Adult Stem Cells

  • Can open his fist and stretch his fingers by himself
  • Can roll from his back onto his side all by himself
  • Can now carry some weight, such as a bottle of apple juice rather than just empty cups
  • Reports improved strength in all his muscles
  • Daily tasks such as eating and writing are easier

Adult Stem Cells “Definitely” and “Absolutely” Worth It

When asked if the trip half way around the world from Wisconsin to China was worth it, Kyle replied “Definitely” and his mother chimed in with “Absolutely.”

From the stem cell article:

Kyle and Penny said they’re happy to have gone through all the fundraising and planning for the treatment that cost more than $30,000, plus expenses.

They’d even consider a second trip in a few years for more injections.

They met families from across the United States, Australia, United Arab Emirates, England and elsewhere during their stay.

It was like one big family,” Kyle said.

Another Spinal Muscular Atrophy Patient Helped By Stem Cell Research

Coincidentally, or probably not so coincidentally, Kyle followed in the footsteps of another Janesville resident with Spinal Muscular Atrophy, Brandon Meinke.

This was a great story because a reporter from the local TV station followed Brandon before and after the stem cell treatment, documenting the improvements.

Brandon received stem cell treatment for his SMA and was able to walk again after the adult stem cells. You can see more on his stem cell therapy here.

original post by Don Margolis

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