The Muscular Dystrophy Association (MDA) has awarded $300,000 over three years to Matthew Disney, professor on the Florida campus of The Scripps Research Institute (TSRI). The new funding will support further work in Disney’s laboratory to optimize two novel drug-like compounds for treating ALS and a form of muscular dystrophy called myotonic dystrophy.
There are more than 30 diseases, including myotonic dystrophy type 1 and ALS (amyotrophic lateral sclerosis or Lou Gehrig’s disease), caused by toxic RNA repeats—errors in the way the genetic code is translated into proteins. These diseases are currently incurable, but Disney’s laboratory is working to develop small molecule drug candidates that can precisely attack and neutralize these toxic repeats, potentially removing the source of the disease from affected cells.
“We want to develop strategies that show there is potential to make drugs that target this class of disease-causing RNAs,” said Disney.
Disney added that, when successful, this approach could open up the field of drug discovery by giving scientists new tools to stop not just myotonic dystrophy type 1 and ALS, but also fragile X syndrome, Huntington’s disease and various ataxias, including spinocerebellar ataxia.
For more information on this research, see Matthew Disney’s Biosketch and the Disney Laboratory Website. The MDA has also published a Q&A with Disney about the work.
The TSRI Career and Postdoctoral Services Office invites you to enroll in a course designed to equip you with the skills, resources and self-assessment information necessary to make informed career choices both inside and beyond academia. Classes will be held Wednesdays, from 12 to 2 PM PST in the California Campus Graduate Office, Seminar Room, and from 3 to 5 PM on the Florida Campus, Room B158, from January 4 to February 15, 2017. For questions or registration information, contact cpso@scripps.edu.
TSRI California faculty, students and staff are invited to help those in need this holiday season by participating in the ScrippsAssists annual Holiday Toy Drive supporting Home Start, a San Diego non-profit agency dedicated to child abuse prevention and family strengthening services.
New and unwrapped toys for children of all ages—many of whom face homelessness or other serious crises—should be dropped off by Monday, December 19, at specially indicated boxes in the lobbies of the Molecular Biology, Immunology, Beckman, DNC and MEM buildings.
For a list of suggested age-appropriate gifts, contact ScrippsAssists project lead Jess Sheu-Gruttadauria at jgruttad@scripps.edu.
To receive consideration at the next TSRI Institutional Biosafety Committee, registration documents must be submitted to Environmental Health and Safety by Friday, December 16, via email to rachellv@scripps.edu. The meeting will be held Wednesday, January 11, 2:30 to 3:30 PM, in the P2 conference room, Building 3301.
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