Research Objective Use gene editing to create tools for the study of mechanisms by which patient-observed mutations lead to myeloproliferative neoplasms. Impact Editing reagents will yield new insight into how…
Translational Candidate Human stem cells in a dish engineered into heart cells to supplement, refine, reduce, and/or ultimately replace human clinical trials. Area of Impact Increase genetic diversity of preclinical…
Translational Candidate In vitro miniaturized array of heart muscle amenable for use in efficient high-throughput drug discovery and screening campaigns. Area of Impact Effective high-throughput screening of drugs on human…
Heart failure, diabetes and neurodegenerative diseases are among the leading causes of death and disability worldwide. These diseases are characterized by the loss of specific cell types and can be…
Heart disease is the number one cause of death in the US. Heart muscle injured during a heart attack does not regenerate, and the resulting damage leads to heart failure,…
Motor neurons degenerate and die as a consequence of many conditions, including trauma to the spinal cord and its nerve roots and degenerative diseases such as amyotrophic lateral sclerosis and…
The Center of Excellence in Stem Cell Genomics will bring together investigators from seven major California research institutions to bridge two fields – genomics and pluripotent stem cell research. The…
Motor neuron (MN) diseases such as spinal muscular atrophy and amyotrophic lateral sclerosis lead to progressive degeneration of MNs, presenting first with muscle weakness, followed by locomotor defects and frequently…
Parkinson’s disease (PD), is one of the leading causes of disabilities and death and afflicting millions of people worldwide. Effective treatments are desperately needed but the underlying molecular and cellular…
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