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MDF Advances DM Research with Fund-a-Fellow Grants

In early May, the Foundation announced a Request for Applications (RFA) for this year's MDF Fund-a-Fellow program. The Fund-a-Fellow program supports our commitment to Care and a Cure for myotonic dystrophy by attracting new investigators to the field of DM research, ultimately advancing DM science and the search for treatments. Two current Fund-a-Fellows, Suzanne Rzuczek, PhD, at The Scripps Research Institute, Florida and Ayal Hendel, PhD, at the Stanford School of Medicine, are in the second year of their Fellowships.  We followed up with them recently to get an update on their MDF-funded research projects.

In the Disney Lab at The Scripps Research Institute, Dr. Rzuczek is investigating the potential of small molecules to disrupt the interaction between repeating CUG RNA and MBNL1 (muscleblind-like 1 protein) using the Disney-optimized spacer. Noting that small molecules are underutilized for targeting RNA, Dr. Rzuczek says, "I see great potential in studying them further as they relate to DM."

Dr. Rzuczek feels a great sense of responsibility as an MDF Fellow. She says, "With each success in the lab I get really excited because I know that those affected by DM, and their family members, depend on researchers like me to find successful therapies." She is pleased with the progress she's making in the lab and is hoping to publish results from her research in the near future.

For now, Dr. Rzuczek says she is uncertain where her career path will lead but says, "I value my experience as an MDF Fellow and my time in the Disney Lab. I hope to continue to make important contributions as a researcher in the coming years."

Dr. Ayal Hendel is at the Stanford School of Medicine and is using engineered nucleases that induce CTG/CAG repeat contractions, possibly correcting the mutation and reducing disease risk. Dr. Hendel has been successful in the laboratory engineering corrected DM1 cells for modeling the disease and is now preparing to use these cells to study different aspects of the disorder. "It is really very promising," he adds.

For Dr. Hendel, the MDF Fellowship has been helpful to his research in many ways, including connecting him with other DM researchers to create opportunities for collaboration. He's looking forward to meeting many of those researchers in person at the 2014 MDF Annual Conference in the fall.

Dr. Hendel hopes to one day have his own lab. "Right now genomic engineering is exciting. Scientific advances in the field are progressing rapidly. I hope to be on the front-line of discovering an effective therapy for DM."

Fund-A-Fellow Awards and Application Process

MDF Fund-a-Fellow grants are $100,000 each, disbursed over two years, and are awarded to post-doctoral students who have received doctoral degrees from accredited US or international institutions within the past three years. Applications will be accepted until 5:00 PM PST Friday, September 5, 2014. Click here for FAF guidelines and the application. 

05/20/2014

 

 

 

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