Our long-term goal is to optimize a cell-based therapy for patients with muscular dystrophy. We are now pursuing this goal by optimizing isolation and engraftment of human muscle stem cells, and by creating new animal models for preclinical testing.
Isolation of muscle progenitors
We are thoroughly comparing the transcriptome--the set of active genes--of human muscle stem cells to that of committed muscle cells using microarray technology to identify specific markers of muscle stem cells that can be used to purify stem cells from primary tissues.
Proliferation of muscle progenitors
We are studying the proliferation mechanisms of muscle progenitors to ultimately generate sufficient muscle cells for therapy.
Ultimately, we seek to optimize engraftment of injected cells by identifying and subsequently boosting the mechanisms by which muscle stem cells fuse to pre-existing myofibers. We also hope to optimize the injection and dissemination of human muscle stem cells in mouse models of muscular dystrophy.
The fibers in our muscles form upon contraction of myotubes (fused muscle cells). Here, a series of myotubes are shown stained with an antibody that detects dystrophin (green), the protein that is functionally altered or lost in the Duchenne-Becker form of the muscle wasting disease, muscular dystrophy.
To read more about our work, visit our lab website.
About Emanuela Gussoni
Emanuela Gussoni is an Assistant Professor of Pediatrics at Harvard Medical School and an Assistant in Medicine at Children's Hospital Boston. She received her PhD in neuroscience from the University of Milan, Italy.
She began developing strategies to treat muscular dystrophy as a postdoctoral fellow in the laboratories of Ferdinando Cornelio, MD, at the Carlo Besta Neurological Institute; Helen Blau, PhD, and Larry Steinman, MD, at Stanford University; and Louis Kunkel, PhD, at Children's Hospital Boston. She started her own laboratory at Children's in 2001.
- Wu MP and Gussoni E. Carbamylated erythropoietin does not alleviate signs of dystrophy in mdx mice. Muscle & Nerve, in press.
- Kang PB et al. Inefficient dystrophin expression after cord blood transplantation in DMD. Muscle & Nerve 2010 Jun; 41(6):746-50.
- Sohn RL et al. A role for nephrin, a renal protein, in vertebrate skeletal muscle cell fusion. Proc Natl Acad Sci USA 2009 Jun 9; 106(23):9274-9.
- Yoon S et al. C6ORF32 is upregulated during muscle cell differentiation and induces the formation of cellular filopodia. Dev Biol 2007 Jan 1; 301(1):70-81.
- Frank NY et al. Regulation of myogenic progenitor proliferation in human fetal skeletal muscle by BMP4 and its antagonist Gremlin. J Cell Biol 2006 Oct 9; 175(1):99-110.