CureDuchenne - Together we Can CureDuchenne
October 2012

Scientists of the Month

October 2012 Newsletter - Scientist of the Month
Left to right: Stanley F. Nelson, M.D.
and M. Carrie Miceli, Ph.D.

M. Carrie Miceli, Ph.D. and Stanley F. Nelson, M.D., Center for Duchenne Muscular Dystrophy at UCLA

Dr. Stanley Nelson and Dr. M. Carrie Miceli are directors of the Center for Duchenne Muscular Dystrophy at UCLA. The Center for DMD supports muscular dystrophy related translational research and clinical care at UCLA.

Dr. Nelson and Dr. Miceli recently received a $6 million grant from the California Institute of Regenerative Medicine (CIRM) to develop a combination therapy for Duchenne muscular dystrophy. The study will use patient-derived stem cells to validate and characterize a combined therapeutic in IND enabling studies.

Dr. Nelson and Dr. Miceli will develop a combination therapy for exon skipping in Duchenne. A drug has been identified through the use of muscle stem cells that can enhance the effectiveness of exon skipping by small pieces of DNA called antisense oligonucleotides (AO) to the DMD gene to restore dystrophin and at least partially correct the defect responsible for loss of muscle function. Dr. Nelson and Dr. Miceli will test the effectiveness of a combination therapy composed of an AO with an FDA-approved drug that boosts its activity, a combination therapy discovered at UCLA.

“The grant allows us to test a combination therapy that has the potential to boost exon skipping activity and make it more effective,” said Dr. Nelson. “Overall, half of Duchenne patients could benefit from exon skipping and this has the potential to broaden the reach to different exons beyond exon 51 (which is furthest along in research).”

The grant provides funding for further preclinical research to test the right dosage range and determine the benefits of the combination drug.

CureDuchenne is proud to have been a sponsor of the Center for Duchenne Muscular Dystrophy for the last five years. And since exon skipping has been an important component of CureDuchenne’s research strategy over the past eight years, we are especially pleased to see this important research taking place at UCLA.

“Our goal at the Center for DMD is to encourage researchers at UCLA to apply their skills to finding new treatments for Duchenne,” said Dr. Miceli. “This strategy is working. Before the Center, we had three labs working on Duchenne and now we have 12 labs. We are helping to accelerate moving the science from the labs into clinical trials.”

Researchers from UCLA are also working on other innovative projects focused on Duchenne such as:

  • Muscle cell membrane stabilization.
  • Stem cell models.
  • High throughput screening.
  • Molecular diagnostic tools for Duchenne.
  • Some Duchenne patients never find out their exactmutation. UCLA researchers are trying to identify contributing genes that makes Duchennemore or less severe in some individuals.

Please contact the Center for DMD at UCLA if you are a Duchenne patient that is interested in donating skin cells. The skin cells can be turned into muscle cells that can be used as a tool in the lab to test andscreen drugs.

Also, please contact the Center if you have an extreme case of Duchenne – very severe or very mild. The Center can be reached at or 310-983-3402.

Powered By Convio