Neurologically impaired mice improve after receiving human stem cells

June 4, 2008

Scientists report a dramatic success in what may be the first documented rescue of a congenital brain disorder by transplantation of human neural stem cells. The research, published by Cell Press in the June issue of the journal Cell Stem Cell, may lead the way to new strategies for treating certain hereditary and perinatal neurological disorders.

Nerve cell projections are ensheathed by a fatty substance called myelin that is produced by oligodendrocytes, a type non-nerve cell in the brain and spinal cord. Myelin enhances the speed and coordination of the electrical signals by which nerve cells communicate with one another. When myelin is missing or damaged, electrical signals are not properly transmitted.

Previous studies have explored the potential utility of cell transplantation for restoring absent or lost myelination to diseased nerve fibers. Much of this research has made use of the ‘shiverer mouse’ animal model which lacks normal myelin and typically dies within months of birth. Yet to date, no transplantation of human neural stem cells or of their derivatives, called glial progenitor cells, have ever altered the condition or fate of recipient animals.

Dr. Steve Goldman and colleagues from the Departments of Neurology and Neurosurgery at the University of Rochester Medical Center, along with collaborators at Cornell, UCLA and Baylor, built on this earlier work by devising a more robust method for the acquisition and purification of human fetal glial progenitor cells.

In addition, they developed a new cell delivery strategy, based on multiple injection sites, to encourage widespread and dense donor cell engraftment throughout the central nervous system of recipient mice. The researchers transplanted human glial stem cells into neonatal shiverer mice that also had a genetically deficient immune system. Immunodeficient mice were used to minimize the rejection of the transplanted cells.

The researchers found that the new transplant procedure resulted in infiltration of human glial progenitor cells throughout the brain and spinal cord. The engrafted mice exhibited robust, efficient and functional myelination. Most notably, many of the mice displayed progressive, neurological improvement and a fraction of the mice were actually rescued by the procedure. “The neurological recovery and survival of the mice receiving transplants was in sharp contrast to the fate of their untreated controls, which uniformly died by five months,” explains Dr. Goldman. Upon histological examination well over a year after the procedure, the white matter of the surviving mice had been essentially re-myelinated by human cells.

“To our knowledge, these data represent the first outright rescue of a congenital hypomyelinating disorder by means of stem or progenitor cell transplantation,” offers Dr. Goldman. “Although much work needs to be done to maximize the number of individuals that respond to transplantation, I think that these findings hold great promise for the potential of stem cell-based treatment in a wide range of hereditary and ischemic myelin disorders in both children and adults.”

Source: Cell Press

Explore further: Researcher explores the current state of domestic animal cloning

Related Stories

Stem cells have more reserves for DNA replication

July 17, 2015

In cell division, nothing is as important as the precise replication of billions of genetic letters that make up DNA. Since this genomic integrity is so fundamental to survival, scientists had assumed that replication mechanisms ...

Can gene editing provide a solution to global hunger?

July 6, 2015

According to the World Food Program, some 795 million people – one in nine people on earth – don't have enough food to lead a healthy active life. That will only get worse with the next global food crisis, predicted to ...

Highly efficient CRISPR knock-in in mouse

May 1, 2015

Genome editing using CRISPR/Cas system has enabled direct modification of the mouse genome in fertilized mouse eggs, leading to rapid, convenient, and efficient one-step production of knockout mice without embryonic stem ...

Recommended for you

Researchers design first artificial ribosome

July 29, 2015

Researchers at the University of Illinois at Chicago and Northwestern University have engineered a tethered ribosome that works nearly as well as the authentic cellular component, or organelle, that produces all the proteins ...

Meet the high-performance single-molecule diode

July 29, 2015

A team of researchers from Berkeley Lab and Columbia University has passed a major milestone in molecular electronics with the creation of the world's highest-performance single-molecule diode. Working at Berkeley Lab's Molecular ...

Playing 'tag' with pollution lets scientists see who's 'it'

July 29, 2015

Using a climate model that can tag sources of soot from different global regions and can track where it lands on the Tibetan Plateau, researchers have determined which areas around the plateau contribute the most soot—and ...

Researchers build bacteria's photosynthetic engine

July 29, 2015

Nearly all life on Earth depends on photosynthesis, the conversion of light energy into chemical energy. Oxygen-producing plants and cyanobacteria perfected this process 2.7 billion years ago. But the first photosynthetic ...

0 comments

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.