Features | Partner Sites | Information | LinkXpress
Sign In
GLOBETECH PUBLISHING LLC
GLOBETECH MEDIA
GLOBETECH PUBLISHING LLC

Combination Gene and Stem Cell Therapy Cures Muscular Dystrophy in Mouse Model

By BiotechDaily International staff writers
Posted on 19 Mar 2013
In a "proof-of-principle" study, researchers have shown that it was possible to cure Duchenne Muscular Dystrophy (DMD) in a mouse model using a combination of gene and stem cell therapy.

Investigators at the University of Minnesota (Minneapolis, USA) began by reprogramming skin cells taken from DMD mice back into an undifferentiated pluripotent stem cell stage. They then employed a gene therapy approach based on the "Sleeping Beauty Transposon" to incorporate the gene for the protein utrophin into the stem cells. Utrophin can substitute for the defective protein dystrophin, which characterizes DMD, without generating an autoimmune response.

The last step was to treat the modified pluripotent cells with the muscle stem cell protein Pax3 (paired box 3), which induced the stem cells to differentiate into muscle cells. Pax3 is expressed in early embryonic growth where it helps to demarcate the paraxial mesoderm. In that way, PAX3 contributes to early striated muscle development since all myoblasts are derived from the paraxial mesoderm.

The modified stem cells were injected into DMD mice. Results published in the March 5, 2013, online edition of the journal Nature Communications revealed that the stem cells were incorporated into muscle tissue that displayed large numbers of micro-utrophin-positive myofibers, with biochemically restored dystrophin–glycoprotein complexes, and improved contractile strength.

"Developing methods to genetically repair muscular dystrophy in human cells, and demonstrating efficacy of muscle derived from these cells are critical near-term milestones, both for the field and for our laboratory," said senior author Dr. Rita Perlingeiro, associate professor of medicine at the University of Minnesota. "Testing in animal models is essential to developing effective technologies, but we remained focused on bringing these technologies into use in human cells and setting the stage for trials in human patients."

"We were pleased to find the newly formed myofibers expressed the markers of the correction, including utrophin," said, Dr. Perlingeiro. "However, a very important question following transplantation is if these corrected cells would self-renew, and produce new muscle stem cells in addition to the new muscle fibers."

Related Links:

University of Minnesota



Channels

Drug Discovery

view channel
Image: Star-like glial cells in red surround alpha-beta plaques in the cortex of a mouse with a model of Alzheimer\'s disease (Photo courtesy of Strittmatter laboratory/Yale University).

Experimental Cancer Drug Reverses Symptoms in Mouse Model of Alzheimer's Disease

An experimental, but clinically disappointing drug for treatment of cancer has been found to be extremely effective in reversing the symptoms of Alzheimer's disease (AD) in a mouse model.... Read more

Biochemistry

view channel
Image:  Model depiction of a novel cellular mechanism by which regulation of cryptochromes Cry1 and Cry2 enables coordination of a protective transcriptional response to DNA damage caused by genotoxic stress (Photo courtesy of the journal eLife, March 2015, Papp SJ, Huber AL, et al.).

Two Proteins Critical for Circadian Cycles Protect Cells from Mutations

Scientists have discovered that two proteins critical for maintaining healthy day-night cycles also have an unexpected role in DNA repair and protecting cells against genetic mutations that could lead... Read more

Business

view channel

NanoString and MD Anderson Collaborate on Development of Novel Multi-Omic Expression Profiling Assays for Cancer

The University of Texas MD Anderson Cancer Center (Houston, TX, USA) and NanoString Technologies, Inc. (Seattle, WA, USA) will partner on development of a revolutionary new type of assay—simultaneously profiling gene and protein expression, initially aiming to discover and validate biomarker signatures for immuno-oncology... Read more
 
Copyright © 2000-2015 Globetech Media. All rights reserved.