Treatment in mice proves effective against Duchenne muscular dystrophy

CHAMPAIGN, Ill. Mice carrying the same gene deficiencies as humans with Duchenne muscular dystrophy experienced dramatic improvements in both their physical condition and life span following an experimental treatment by researchers at the University of Illinois.

By enhancing the production of a naturally occurring molecule on muscle tissue, the scientists reduced muscle-related problems and increased by three-fold the lifetimes of affected mice. Their work appears in the March 19 issue of the Journal of Cell Biology.

The work suggests that a gene therapy or a pharmaceutical approach targeting the molecule may be possible for human treatment, said Stephen J. Kaufman, a professor of cell and structural biology and of the College of Medicine. "The implications are that you could do gene therapy with an integrin chain to treat a muscular dystrophy thats caused by a membrane protein deficiency," he said. "Or you could chemically stimulate integrin chain production from the patients existing integrin chain genes."

Kaufmans lab discovered the molecule in question the alpha 7 integrin in 1985. A deficiency of this molecule exists in several forms of congenital muscular dystrophy. Conversely, Kaufman and his colleagues found that more of the integrin is present in Duchenne patients. These patients fail to produce another protein, dystrophin, which muscles also require for structural and functional integrity.

This discovery led to the idea that excess integrin may compensate for the lack of dystrophin and another similar protein, utrophin. To test their hypothesis, Kaufmans team used mice that did not produce dystrophin or utrophin, and they engineered them to produce even more of the alpha 7 integrin protein. Untreated mice developed debilitating muscular dystrophy, suffered severe weight loss and 50 percent died before reaching 12 weeks of age. Mice with enhanced alpha integrin production did not suffer severe muscular problems, maintained good m

Contact: Jim Barlow
University of Illinois at Urbana-Champaign

Page: 1 2

Related biology news :

1. St. Petersburg/New Haven Partnership for HIV/AIDS Care, Treatment and Support launched
2. Treatment stimulates growth and regeneration of injured adult skeletal muscle
3. Treatment of lupus in mice may point the way to human treatment
4. Treatment protects mouse ovaries from radiation therapy
5. National Institute on Drug Abuse launches National Drug Addiction Treatment Clinical Trials Network
6. Treatment restores normal heart rhythm in patients
7. Researchers Develop New Treatment For Hepatitis B And C As Nation Anticipates Surge In Number Of Cases
8. Researchers: Exciting Discovery Might Improve Cancer Treatment
9. Coffee Without Addiction, Possible New Treatment For Osteoporosis, Wrinkle-Free Cotton, And More
10. Scientists Discover Molecular Trigger Of Mucus Production, Suggesting First Treatment For Deadly Bronchitis, Other Diseases
11. Wistar Scientists Define Mechanism Of Action Used By Potential Magic Bullet In Cancer Treatment

Post Your Comments:

(Date:6/19/2020)... ... June 18, 2020 , ... Join experts from Envigo ... in a live webinar on Wednesday, July 8, 2020 at 11am EDT ... we genetically engineer new models. However, while modifying the genome may be more straightforward, ...
(Date:6/11/2020)... ... June 09, 2020 , ... Superior Controls, Inc. ... automation and IT solutions for the life sciences industry, is pleased to announce the ... working out of the company’s Seabrook, New Hampshire office. In his new role, ...
(Date:6/5/2020)... ... ... Greffex, a pioneering vaccine and gene therapy company based in Houston, Texas, welcomes ... a global perspective to make an impact in a post pandemic world,” said Co-Chairman ... independent, director of the company. , “The Greffex board is excited to have Neil ...
Breaking Biology News(10 mins):
(Date:7/1/2020)... , ... June 29, 2020 , ... ... to date growth of 40% in 2020, despite many obstacles created as a ... aims to increase revenue for its partnered medical practices and medical manufacturers by ...
(Date:6/28/2020)... ... June 25, 2020 , ... The Indoor Lab, a leading provider of sensor ... of all types to ensure cleanliness, distancing and occupancy with its patent pending WipeAway™ ... enabled business will provide a health performance index system that scores all aspects of ...
(Date:6/28/2020)... ... June 26, 2020 , ... The Security Industry ... its strong opposition to the recently introduced bicameral Facial Recognition and Biometric ... most federal use of nearly all biometric and related image analytics technologies, incorrectly ...
(Date:6/23/2020)... ... 2020 , ... Regen Suppliers, the one stop shop for ... get the fourth unit free with no limit on the amniotic derived exosomes. ... have been a game changer for regenerative therapies. The products are manufactured at ...
Breaking Biology Technology:
Cached News: