Research Objective This proposal will develop a cell-based therapy that can restore vision in retinal degeneration associated blindness such as Stargardt disease and age-related macular degeneration. Impact Chemically induced method…
Research Objective An intravenously injectable virus that converts the scar cells responsible for liver cirrhosis into the cells that provide most of the liver’s function, thereby preventing or reversing liver…
Research Objective To develop a gene therapy product to deliver cardiac reprogramming factors into the heart for regeneration of new heart muscle. Impact The proposed candidate would regenerate heart muscle…
Research Objective We will generate human airway stem cells by direct transcriptional reprogramming of fibroblasts. We will use these induced airway stem cells to model motile cilia disease in a…
We and other groups have recently shown that it is possible to convert skin cells from foreskin of newborns into nerve cells that closely resemble nerve cells of the brain…
The current roadblocks to hematopoietic stem cell (HSC) therapies include the rarity of matched donors for bone marrow transplant, engraftment failures, common shortages of donated blood, and the inability to…
Many human diseases and injuries that affect the brain and nervous system could potentially be treated by either introducing healthy neurons or persuading the cells that normally provide supporting functions…
Recently, we devised and reported a new regenerative medicine paradigm that entails temporal/transient overexpression of induced pluripotent stem cell based reprogramming factors in skin cells, leading to the rapid generation…
Heart disease is a leading cause of mortality. The underlying pathology is typically loss of heart muscle cells that leads to heart failure. Because heart muscle has little or no…
Although the liver can regenerate itself, chronic or overwhelming damage can cause life-threatening liver failure. Currently, the only therapy for liver failure is liver transplantation. Because the supply of cadaveric…
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