Understanding your most important tissue

What governs development, disease & regeneration of the heart & other muscles?

We are seeking to delineate the mechanisms that govern development, disease and regeneration of the heart and other muscles and to build upon this knowledge to restore muscle function during disease and aging.

As one approach toward this goal, we are applying CRISPR/Cas9-mediated genome editing to promote muscle repair in Duchenne muscular dystrophy (DMD), which is caused by mutations in the dystrophin gene.

We have established new cellular, genetic, and biochemical tools, as well as novel strains of genetically modified mice, for optimizing DMD gene correction in vivo and in human muscle cells derived from iPS cells of DMD patients.

We have also discovered a new myogenic stem cell marked by the expression of the Twist2 transcription factor. Twist2-positive myogenic progenitors and morphologically and molecularly distinct from muscle satellite cells and display potent regenerative activity for adult muscle, providing a new inroad into muscle regeneration and repair.

Finally, through CRISPR screening for genes required for muscle and heart growth and development, we have uncovered new genes with key roles in these processes.

The opportunities and obstacles in the path toward regeneration and permanent correction of diseases of muscle and the heart will be discussed.

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