- Inside UNM
Cripps studying muscle development and limb-girdle dystrophy
Biology Professor Richard Cripps is a big fan of abba. No not the Swedish pop band from the '70s, but a gene found in fruit flies. Cripps' abba could turn out to make a bigger splash than even the legendary Swedish singers did. Mutations or flaws in the human version of the abba gene cause one form of limb-girdle muscular dystrophy (LGMD). Cripps' experiments, funded with a $339,561 grant from the Muscular Dystrophy Association, will define at the molecular level how the protein made by abba functions in fruit fly muscles, and should provide valuable insights into how the comparable gene works in humans.
The fruit fly is frequently used as a model organism for research because the mechanisms of muscle development in this animal are remarkably similar to those of vertebrates, and the genetic processes are easier to study in flies.
"It is critical to understand what is going on at the molecular level of a disease," says Valerie Cwik, M.D., MDA executive vice president for research and medical director. "Pathways to therapies for limb-girdle and other muscular dystrophies are complex, so the studies Cripps' team is conducting on muscle function are vital to our push to develop therapeutics."
Cripps is one of 38 research leaders receiving new multi-year awards from MDA. His three-year project is part of the $14.1 million in new research funding approved during the Association's July Board meeting. This is Cripps' fourth MDA grant. He has received more than $787,000 from MDA to advance his research. To learn more about Cripps and his current project, listen to his recent MDA Q&A; podcast.
"One of the ah-ha moments was when we realized that the fly dystrophic mutation was probably in the same gene (that) affects human muscle. That was a biggie," said Cripps. "Reproducing the dystrophic disease state in fly muscle using RNAi technology (a recently developed technology that enables researchers to switch off individual genes) was another big breakthrough. We expect many more of these strides in the coming years, thanks to the support the project is receiving from MDA."
"MDA has continually succeeded in driving the field forward by supporting imaginative, cutting-edge biomedical research,'' Cripps said. "By allowing many ambitious scientific initiatives to gain a strong foothold, MDA enables researchers to lay firm groundwork for large-scale research to gain priority public funding from the NIH (National Institutes of Health). MDA already has made an indelible impact on the rate at which the patients of degenerative muscle disorders will be able to expect new and effective treatments in the future."
Research grant applications are peer-reviewed twice yearly by MDA's Medical and Scientific Advisory Committees, comprising world-renowned experts in neuromuscular disease research. The most promising of some 500 applications received each year are recommended for funding to the MDA Board of Directors.
According to R. Rodney Howell, M.D., chairman of the MDA Board of Directors, "Generous Americans responding to the annual Jerry Lewis MDA Telethon, and to thousands of other special events benefiting the families served by MDA, deserve much of the credit for the rapid progress being made toward treatments for neuromuscular diseases. It's their strong belief in MDA's capable stewardship of public funds that's enabling so much to be accomplished — even in a sluggish economy."
Often credited for its leadership in building the field of neuromuscular disease research, MDA also enhanced clinical care for individuals affected by muscle disorders, achieving important quality of life and longevity gains. The Association, which has invested almost $39 million in 2010 in research worldwide, is the first nonprofit to earn a Lifetime Achievement Award from the American Medical Association ("for significant and lasting contributions to the health and welfare of humanity.")
MDA-funded scientists have uncovered the genetic defects that cause several forms of muscular dystrophy; Charcot-Marie-Tooth disease (CMT); a form of amyotrophic lateral sclerosis (ALS, or Lou Gehrig's disease); childhood spinal muscular atrophy (SMA) and other neuromuscular conditions. Now entering a period of increasing numbers of clinical trials of potential therapeutics, the Association's network of approximately 200 hospital-affiliated clinics is instrumental in identifying appropriate candidates for clinical trials, and in helping to refine outcome measures for those clinical trials.
More than 1 million people in America are affected by neuromuscular diseases. Residents of New Mexico who are affected by any of the more than 40 muscle diseases in MDA's program can receive excellent medical care at the MDA Clinic at the University of New Mexico Hospital. Individuals living with ALS are encouraged to visit the MDA/ALS Center at the University of New Mexico Health Sciences Center.
MDA is the nonprofit health agency dedicated to curing muscular dystrophy, ALS and related diseases by funding worldwide research. View video of MDA research at MDA research video. The Association also provides comprehensive health care and support services, advocacy and education. For more information visit: MDA research and programs.
For more information about these new grants, visit MDA's "Grants at a Glance" Grants at a Glance, an online slideshow that showcases each grant with photos and detailed information.
Media contacts: MDA, Jim Brown (520) 529-5320; e-mail: email@example.com or UNM, Steve Carr (505) 277-1821; e-mail: firstname.lastname@example.org