Since their inception, the Wellstone Centers have contributed to over 800 publications spanning basic, translational, and clinical research. Examples of recent Wellstone Center research advances include:
- potential therapeutics for facioscapulohumeral muscular dystrophy (FSHD);
- novel gene editing approaches to target cardiac muscle and other genetic mutations in Duchenne muscular dystrophy (DMD); and
- better methods to assess cognitive symptoms in myotonic dystrophy type 1 (DM1) and respiratory symptoms in DMD.
Research supported by Wellstone Centers has led to multiple FDA approved treatments for muscular dystrophies. Notably, the Wellstone Center at the Children's National Medical Center in Washington, D.C., which received funding from 2005 to 2010, contributed to the development of antisense oligonucleotide exon-skipping therapies. These innovative treatments have since gained FDA approval for specific subtypes of DMD.
Research at the UT Southwestern Wellstone Center has advanced toward the translation of therapeutic genome editing for DMD, through the successes of CRISPR-Cas9-mediated genome editing in correcting genetic mutations in DMD animal models, as well as recent advances in the delivery of those corrections.
Research at the University of Washington Wellstone Center and a former Center at the University of North Carolina contributed to the development of shortened forms of the dystrophin gene and improvements in viral gene therapy vectors. These advances helped facilitate the development and recent FDA approval of Elevidys, which is the first gene therapy for DMD.