Stem cells for Duchenne?

Adult stem cells taken from humans suffering from Duchenne muscular dystrophy can be genetically modified and used to treat the disease in a mouse model, researchers linkurl:report;http://www.cellstemcell.com/ today in Cell Stem Cell. Duchenne muscular dystrophy is a progressive condition caused by a mutation on the X chromosome that leads to a lack of dystrophin protein in muscle. The mutation is usually caused by a deletion or mutation in the gene, leading to a shift in the reading frame of m

| 1 min read

Register for free to listen to this article
Listen with Speechify
0:00
1:00
Share
Adult stem cells taken from humans suffering from Duchenne muscular dystrophy can be genetically modified and used to treat the disease in a mouse model, researchers linkurl:report;http://www.cellstemcell.com/ today in Cell Stem Cell. Duchenne muscular dystrophy is a progressive condition caused by a mutation on the X chromosome that leads to a lack of dystrophin protein in muscle. The mutation is usually caused by a deletion or mutation in the gene, leading to a shift in the reading frame of mRNA translation. In past studies, injecting Duchenne mice with normal muscle cells has temporarily staved off disease symptoms, but that technique does not work reliably and can cause immune rejection. So researchers have linkurl:proposed;http://www.ncbi.nlm.nih.gov/sites/entrez?Db=PubMed&Cmd=ShowDetailView&TermToSearch=16691118&ordinalpos=3&itool=EntrezSystem2.PEntrez.Pubmed.Pubmed_ResultsPanel.Pubmed_RVDocSum delivering muscle progenitor cells instead. Yvan Torrente of the University of Milan, Italy, and colleagues took human blood- and muscle-derived stem cells from Duchenne patients. They used linkurl:antisense oligonucleotides;http://www.the-scientist.com/article/display/21438/ delivered by linkurl:lentiviral vectors;http://www.the-scientist.com/article/display/19298/ to mask the incorrect mRNA splicing sites, thus returning translation to its normal reading frame. They then intramuscularly injected the corrected cells into the sick mice. Within three weeks, the transplanted cells gave rise to muscle fibers and spurred production of the dystrophin protein. The authors caution, however, that using viral vectors may cause tumor formation, and that details of the technique such as its efficiency remain to be worked out. Both the antisense techniques and muscle cell transplantation have been tried before in Duchenne, note linkurl:Kay Davis;http://www.hertford.ox.ac.uk/main/content/view/155/249/ of Oxford University and linkurl:Miranda Grounds;http://school.anhb.uwa.edu.au/personalpages/grounds/ of the University of Western Australia in an accompanying review article. But the combination of the two makes this a proof of principle study that shows that "steady progress is being made toward the goal of stem cell-mediated restoration of dystrophin expression," they write.
Interested in reading more?

Become a Member of

The Scientist Logo
Receive full access to more than 35 years of archives, as well as TS Digest, digital editions of The Scientist, feature stories, and much more!
Already a member? Login Here

Meet the Author

  • Alla Katsnelson

    This person does not yet have a bio.
Share
Image of a woman in a microbiology lab whose hair is caught on fire from a Bunsen burner.
April 1, 2025, Issue 1

Bunsen Burners and Bad Hair Days

Lab safety rules dictate that one must tie back long hair. Rosemarie Hansen learned the hard way when an open flame turned her locks into a lesson.

View this Issue
Conceptual image of biochemical laboratory sample preparation showing glassware and chemical formulas in the foreground and a scientist holding a pipette in the background.

Taking the Guesswork Out of Quality Control Standards

sartorius logo
An illustration of PFAS bubbles in front of a blue sky with clouds.

PFAS: The Forever Chemicals

sartorius logo
Unlocking the Unattainable in Gene Construction

Unlocking the Unattainable in Gene Construction

dna-script-primarylogo-digital
Concept illustration of acoustic waves and ripples.

Comparing Analytical Solutions for High-Throughput Drug Discovery

sciex

Products

Atelerix

Atelerix signs exclusive agreement with MineBio to establish distribution channel for non-cryogenic cell preservation solutions in China

Green Cooling

Thermo Scientific™ Centrifuges with GreenCool Technology

Thermo Fisher Logo
Singleron Avatar

Singleron Biotechnologies and Hamilton Bonaduz AG Announce the Launch of Tensor to Advance Single Cell Sequencing Automation

Zymo Research Logo

Zymo Research Launches Research Grant to Empower Mapping the RNome