Features | Partner Sites | Information | LinkXpress
Sign In
JIB
GLOBETECH PUBLISHING
BioConferenceLive

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



comments powered by Disqus

Channels

Drug Discovery

view channel
Image: Molecular rendering of the crystal structure of parkin (Photo courtesy of Wikimedia Commons).

Cinnamon Feeding Blocks Development of Parkinson's Disease in Mouse Model

A team of neurological researchers has identified a molecular mechanism by which cinnamon acts to protect neurons from damage caused by Parkinson's disease (PD) in a mouse model of the syndrome.... Read more

Therapeutics

view channel
Image: This type of electronic pacemaker could become obsolete if induction of biological pacemaker cells by gene therapy proves successful (Photo courtesy of Wikimedia Commons).

Gene Therapy Induces Functional Pacemaker Cells in Pig Heart Failure Model

Cardiovascular disease researchers working with a porcine heart failure model have demonstrated the practicality of using gene therapy to replace implanted electronic pacemakers to regulate heartbeat.... Read more

Lab Technologies

view channel

Precise Ion Irradiation Dosing Method Developed for Cancer Therapy

Scientists are employing nuclear physics principles to provide more effective approaches to radiotherapy treatment for cancer patients. Radiation therapy using heavy ions is best suitable for cancer patients with tumors that are difficult to access, such as in the brain. These particles scarcely damage the penetrated... Read more

Business

view channel

Cancer Immunotherapy Sector Predicted to Surge to USD 9 Billion Across Major Pharma Through 2022

The immunotherapy market will experience substantial growth through 2022, increasing from USD 1.1 billion in 2012 to nearly USD 9 billion in 2022 (corresponding to 23.8% annual growth) in the United Kingdom, United States, France, Germany, Italy, Spain, and Japan, according to recent market research. This notable growth... Read more
 
Copyright © 2000-2014 Globetech Media. All rights reserved.