Projects

Development of Neuromuscular Junctions and Disease Models in a Dish for ALS

Motor neuron diseases (MNDs) are classified in four main groups in which amyotrophic lateral sclerosis (ALS) represents the most common...

Evaluation of muscle satellite cell targeting

Correcting genetic defects within the endogenous muscle SCs could provide a long-term therapy for muscle disorders with potential to repair...

Gene editing with CRISPR/Cas9 for Duchenne Muscular Dystrophy

CRISPR/Cas9 technology has the ability for personalized and permanent gene correction.  Using CRISPR/Cas9, we developed a platform to remove a...

Maturation of human pluripotent stem cells towards adult skeletal muscle and muscle stem cells

The developmental status of human pluripotent stem cell (hPSC) derived skeletal muscle cells is not well defined. By using directed...

Tools and Technology Development for Pluripotent Stem Cells and Differentiation

We have several projects aimed as using tailored engineering platforms to enhance our ability to reprogram, expand and/or differentiate hPSCs...

Transplantation of hPSC-derived skeletal muscle progenitor cells for muscular dystrophy therapies

The functional and therapeutic potential of human fetal, adult, and hPSC derived skeletal muscle progenitor cells (SMPCs) in vivo is...

Understanding Human Developmental Somitogenesis and Myogenesis

Somites form during embryonic development and give rise to various cell and tissue types, such as skeletal muscles, ribs, vertebrae,...

FUNDING

California Institute of Regenerative Medicine