Nature Reports Stem Cells
Published online: 10 January 2008 | doi:10.1038/
Dystrophy patients' muscle stem cells treat mice
Hannah Hoag1
Cells modified to correct a mutation help muscles grow, function
Duchenne muscular dystrophy (DMD) is one of the most common lethal
genetic disorders in boys. Mutations in the dystrophin (DMD) gene
cause progressive muscle wasting and lead to paralysis and death.
Researchers have long hoped to restore dystrophin function by
inserting a working copy of the gene into muscle cells. But at more
than 2.5 million base pairs, it is one of the longest genes in the
human genome and one of the most difficult to manipulate.
In the December issue of Cell Stem Cell1, researchers led by Yvan
Torrent, from the University of Milan, and Luis Garcia, at the French
National Institute of Health and Medical Research (INSERM), report
that a genetic patch can repair the defective dystrophin gene in
adult human stem cells. Moreover, when mice with DMD are injected
with these cells, their muscles grow and function better. The
findings suggest that one day doctors may be able to treat the
disease using a patient's own cells, reducing the possibility of
rejection.
Dystrophin gene mutations generate a transcript that cannot be
translated into a protein. In the Cell Stem Cell paper, the
researchers used antisense oligonucleotides to mask the mutations
responsible for the defective transcript. (More specifically, they
used antisense RNA sequences that caused the cellular machinery to
skip problematic exons within the gene.) Although the resulting
transcript differs from the normal gene variant, it can still produce
a functional protein.
The researchers collected adult muscle stem cells from boys with DMD,
modified those cells with their genetic patch, and injected them into
the arteries and muscles of dystrophic mice. The transplanted stem
cells produced functional human dystrophin.
The group is currently testing this approach in dogs, which are
subject to a condition that more closely resembles human DMD, to see
if the therapy improves their gait, but clinical trials are still a
ways off.
Caution is warranted. Last year a study by other researchers found
that dogs with muscular dystrophy could be helped by injections of a
type of stem cell called a mesoangioblast, scraped from the blood
vessels of normal dogs. Injecting genetically corrected cells from
the diseased dogs had little effect, possibly because the researchers
used a shorter version of the gene that was easier to work with2. A
recent Brief Communication in Nature questioned how well the
treatments using cells from normal dogs worked3.
Still, a dual approach of using both cell and gene-based therapies
has several advantages, says Garcia. "It brings new cells into a
system that needs new progenitors.
cells because they belong to the patient."
Related Articles
Stem cells treat wasted muscles
Top of pageReferences
Benchanouir, R. et al. Restoration of human dystrophin following
transplantation of exon-skipping-
into dystrophic mice. Cell Stem Cell 1, 646–657 (2007).
Sampaolesi, M. et al. Mesoangioblast stem cells ameliorate muscle
function in dystrophic dogs. Nature 444, 574–579 (2006). | Article |
PubMed | ISI | ChemPort |
Bretag, A. Stem cell treatment of dystrophic dogs. Nature 450, E23,
doi:doi: 10.1038/nature06437 (2007). | Article |
Author affiliation
Hannah Hoag is a science journalist based in Montreal.
http://www.nature.
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