Regenerative medicine with mRNA to rejuvenate aging cells

Studying the muscle stem cells in boys suffering fromDuchenne muscular dystrophy, Stanford scientist Helen Blau found that their telomeres–the protective caps at the end of chromosomes–were unusually short. Now, in a new study, Blau and a small group of colleagues say that they have developed a method to use modified messenger RNA to lengthen telomeres, opening a pathway to countering the effects of aging in cells–a process that might apply to a range of diseases from DMD to diabetes and heart disease.

Telomeres naturally shorten every time a cell divides, until the cell naturally stops dividing and dies. But the new approach to extending the telomeres could turn back the clock on cells’ lifespan, which would have a profound impact on drug discovery and testing.

“Now we have found a way to lengthen human telomeres by as much as 1,000 nucleotides, turning back the internal clock in these cells by the equivalent of many years of human life,” said Helen Blau, a professor of microbiology and immunology at Stanford. “This greatly increases the number of cells available for studies such as drug testing or disease modeling.”

To accomplish this, the team turned to the enzyme telomerase, which is used to keep telomeres in stem cells in top condition, according to the Stanford story. They developed a method that is also only temporary, allowing the scientists to contain the impact to prevent the kind of uncontrolled division associated with cancer metastasis. After several applications, the telomeres found in human muscle and skin cell were extended by about 10%. The process spurred cell divisions for both and their RNA approach was able to work without triggering an immune response.

“This study is a first step toward the development of telomere extension to improve cell therapies and to possibly treat disorders of accelerated aging in humans,” said Dr. John Cooke. Cooke, a co-author of the study, was formerly a professor of cardiovascular medicine at Stanford. He is now chair of cardiovascular sciences at the Houston Methodist Research Institute.

It’s obviously very early days for this research project, but the researchers are thinking big about its potential.

“One day it may be possible to target muscle stem cells in a patient with Duchenne muscular dystrophy, for example, to extend their telomeres,” says Blau. “There are also implications for treating conditions of aging, such as diabetes and heart disease. This has really opened the doors to consider all types of potential uses of this therapy.”

http://www.fiercebiotechresearch.com/story/stanford-scientists-use-modified-rna-rejuvenate-aging-cells/2015-01-27