MDF Receives $1M Award to Drive Gene Editing Technologies Targeting DM1

MDF has been awarded a $1M donation by an anonymous family in the DM community to launch development of gene editing technology to pursue the search for a cure for DM1. The approach is to foster early stage gene editing projects in research laboratories that will identify the most practical and feasible strategies to move forward into clinical testing. MDF has conducted a review of the state of the science and identified leadership in the field, and is now developing an expert workshop to be held in early 2018 to lay out guidance and a final road map for the gene editing project. Grantees will then be determined via an open and competitive application process.

CRISPR & Beyond

Gene editing technology is evolving rapidly. Many of our community members may be aware of CRISPR, since it has received considerable coverage in both scientific and lay media and has been featured multiple times in MDF’s Dispatch and Research News. CRISPR is only one recent gene editing strategy being examined for its potential to correct errors in genes that lead to disease. One potential approach is to utilize gene editing to remove the expanded CTG repeats from the DMPK gene, thereby halting production of toxic RNA and the downstream symptoms of DM1. The upcoming MDF gene editing workshop will seek out expert input on the optimal strategy and best practices to guide this program.

Significant Opportunity for Research Leadership

The $1M donation to launch this program is one of the most significant single donations MDF has ever received, and presents an incredible opportunity for MDF to initiate and drive investigation of gene editing approaches as possible opportunities to develop a cure for DM. The project should also significantly enhance the information available regarding opportunities to use gene editing technology to pursue therapies for DM. MDF looks forward to keeping the community apprised of progress on this groundbreaking program as it moves forward.