Potential Cure for Muscular Dystrophy
(Ivanhoe Newswire) – Scientists may have found a way to counteract the muscle degeneration caused by muscular dystrophy by boosting the activity of a vitamin-sensitive cell adhesion pathway.
The discovery is particularly important for congenital muscular dystrophies, which are progressive, debilitating and often lethal diseases that currently remain without a cure. Researchers found that they could improve muscle structure and function in a zebrafish with muscular dystrophy by supplying a common cellular chemical (or its precursor, vitamin B3) to activate a cell adhesion pathway.
The study was led by UMaine Associate Professor of Biological Sciences, Clarissa Henry. The researchers discovered that a pathway involving a common cellular chemical called nicotinamide adenine dinucleotide (NAD+) plays a role in the formation of organized basement membranes in muscle tissue, during development of the fish embryo. As disordered basement membranes are seen in many different types of muscular dystrophies, the researchers wondered whether activating this pathway might reduce the severity of some muscular dystrophies. The researchers show that NAD+ improves the organization of laminin in a zebrafish with muscular dystrophy. Zebrafish lacking either of the two main receptors for laminin have a disorganized basement membrane, causing muscle degeneration and difficulties with movement. However adding extra NAD+, or even a vitamin packet containing vitamin B3 (niacin, a precursor to NAD+), significantly reduced these symptoms.
The research team found that the main protective effects of NAD+ come from enhancing the organization of the laminin structure in the basement membrane, which helps to increase the resilience of diseased muscle fibers. Because the same cell adhesion complexes are found in humans, the research team is optimistic that these findings may one day positively impact patients with muscular dystrophies. "Although there is a long way to go, I'm hopeful that our data could eventually lead to new adjuvant therapies," University of Maine Ph.D. student Michelle Goody, who led the research team with Prof. Henry, was quoted as saying. "One of my favorite aspects of this study is that it is a poster child for how asking basic biological questions can lead to exciting discoveries that may have future therapeutic potential,” Prof. Henry was quoted as saying.
Source: PLOS Biology, October 2012