GENETIC ENGINEERING OF SKELETAL MUSCLE CELLS FOR AUTOLOGOUS CELL TRANSPLANTATIONS  
UCLA Technology Available For Licensing

UCLA physiologists have invented a method that enables adult skeletal cells to functionally resemble heart muscle cells. Using this method, they have successfully engineered mouse adult skeletal muscle fibers to demonstrate electrophysiological features of human cardiac fibers. This method could clear a major roadblock for the medical applications of skeletal myoblasts in post-infarction treatments.

BACKGROUND:  Heart disease is the No.1 killer of Americans today, responsible for 1 of every 5 deaths in 2004. There were 1,200,000 new and recurrent cases of myocardial infarction (or heart attack) per year. More importantly, about 38% of people who experience a heart attack will die from it. The options of post-infarction treatments are very limited. The extreme shortage of healthy heart donors and common immunological complications after heart transplantation make heart transplantation unfeasible. Autologous cell transplantation is a promising alternative approach, however, the success of this treatment hinges on the search for ideal sources of donor cells. Currently, two types of cells, pluripotential stem cells and skeletal myoblasts, have been considered. Skeletal myoblasts have several advantages over pluripotential stem cells, such as the ease to obtain them, resistance to the ischemic conditions and no possible tumorigenicity. Nevertheless, a major drawback of using skeletal myoblasts is that these cells are inefficient in engaging in the mechanical performance of the surrounding heart tissue, leading to arrhythmias (or abnormal heart rhythms).

INNOVATION:  Researchers at UCLA have invented a way to genetically modify skeletal myoblasts prior to cell transplantation and enable modified cells to demonstrate electrophysiological features essential for normal heart functions. This invention has resolved the deficiency of skeletal myoblasts in cardiac functions after autologous cell transplantation, and could clear a major roadblock for the medical applications of skeletal myoblasts in post-infarction treatments.

POTENTIAL APPLICATIONS 

ADVANTAGES

DEVELOPMENT-TO-DATE:  UCLA researchers have genetically engineered mouse skeletal muscles to demonstrate the electrophysiological features of human heart muscles in mice in vivo.

Related Papers (Selected)

Reference: UCLA Case No. 2008-235

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availability, please contact the following UCLA office:

UCLA Office of Intellectual Property
11000 Kinross Avenue, Suite #200
Los Angeles, CA 90095
Tel: 310-794-0558 Fax: 310-794-0638
email: ncd@research.ucla.edu
NCD URL:   http://www.research.ucla.edu/tech/ucla08-235.htm

Lead Inventor: Julio L. Vergara

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Copyright © 2009 The Regents of the University of California.

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