Hereditary Tyrosinemia Type 1 (HT1) is a genetic disorder that can lead to liver and kidney failure, and is fatal without treatment. There is no cure. This project aims to develop a lentivirus-mediated gene therapy to treat multiple inborn errors of liver metabolism, with the research in HT1 serving as a model for future studies of other rare liver diseases.

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Cardiovascular disease is the most common cause of death in the United States, and many patients need heart transplants to survive. Currently, heart transplantation is limited based on the scarcity of donor organs. This proposal aims to see if it is possible to develop humanized and personalized hearts in gene-edited animals.

The decline of memory and thinking processes seen in aging, neurodegenerative diseases (like Alzheimer’s disease), and exposure of the brain to radiation, are believed to be caused in part by senescence (gradual deterioration) of stem cells in the brain. This study examines if selectively allowing these deteriorated cells to die will combat damage to the central nervous system, and if it is safe and effective.

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Millions of U.S. citizens suffer from vein valve insufficiency, often resulting in chronic leg ulcers that cause pain, immobility, and a reduced quality of life. The goal of this research is to develop tissue-engineered vein valve, an “off-the-shelf” implantable device. If successful, it will be the first prosthetic vein valve available to treat suffering from ulcers due to chronic venous insufficiency.

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Spinocerebellar ataxia type 1 (SCA1) is a fatal neurodegenerative disease with no available therapies. This proposal will determine therapeutic potential of a brain-derived neurotrophic factor (BDNF) to regenerate communication between cells in the brain, prevent neuronal loss, and delay disease onset and progression in SCA1. Completion of this research will provide a foundation for developing preventive and therapeutic interventions for SCA1 and other neurodegenerative diseases.

The goal of this project is to develop microcapsules that could be used as cell carriers during differentiation of stem cells into pancreatic islets. These same capsules may also be useful as vehicles for islet transplantation. Developing this technology will enable better scale-up of islet production from stem cells, may result in cost reduction, and may also alleviate problems associated with immune rejection of transplanted islets.

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This clinical trial investigates whether adipose-derived (fat tissue-derived) mesenchymal stem cells can be safely administered into the cerebrospinal fluid of patients with traumatic spinal cord injury and what the effects this may have on functional improvement, activities of daily living, and quality of life. If successful, this study may create a viable clinical option for spinal cord injury patients by establishing feasibility and safety data for patients who do not have many treatment options.

Many regenerative therapies are based on using a patient’s own cells to create induced pluripotent stem (iPS) cells that can then be differentiated (transformed) into other cell types that the patient needs. This investigator’s previous research found that differentiation of human iPS cells to cardiomyocytes is regulated by circadian rhythms (like our internal body clock). This project hopes to establish a new way to accelerate the differentiation process, which will allow treatments to reach patients faster.

The goals of this project are to test the initial safety and feasibility of RECLAIM, a single stage cartilage repair technique using the patient’s own cartilage cells and allogeneic mesenchymal stem cells.  The study will assess patient’s improvement in pain and function following the procedure, while actively monitoring safety and adverse events.