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UConn Health Cell and Genome Sciences
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2008 State Stem Cell Funding

$450,000 – Directed Differentiation of ESCs into Cochlear Precursors for Transplantation as Treatment of Deafness.

Kent Morest, M.D., UConn Health.

This research lays the foundation for stem cell therapy for human hearing disorders, including deafness, partial hearing loss and tinnitus, or ringing in the ears, which can result from noise, drugs, infections and aging. The research is exploring the potential to create new sensory neurons and their connections from embryonic stem cells.


$450,000 – Human Embryonic Stem Cells as a Source of Radial Glia, Neurons and Oligodendrocytes.

Nada Zecevic, Ph.D., UConn Health.

The project addresses current scientific limitations on the ability to characterize the progenitor cells that develop into the principal components of the brain and nervous system, neurons and their surrounding, supportive tissue, made up of astrocytes and oligodendrocytes. Oligodendrocytes produce the myelin sheaths that protect nerves, which are damaged in multiple sclerosis, so this research is of special clinical interest in that respect. Better understanding development of the brain is essential in order to use stem cells to treat many neurodegenerative diseases.


$450,000 – Synaptic Replenishment Through Embryonic Stem Cell-derived Neurons in a Transgenic Mouse Model of Alzheimer's Disease.

Ben Bahr, Ph.D., UConn.

There is great interest in the medical applications of human stem cells to treat age-related diseases like Alzheimer's. However, little is known about the ability of stem cells to survive once they are transplanted into the aged brain. Some studies have shown a high level of cell death within days of implantation. This research explores the processes that contribute to poor transplant survival rates and classes of drugs that can enhance survival rates of transplanted stem cells.


$450,000 – Modeling Motor Neuron Degeneration in Spinal Muscular Atrophy Using Human Embryonic Stem Cells.

Xue-Jun Li, Ph.D., UConn Health.

Survival motor neuron (SMN) protein is produced thanks to a gene called the survival motor neuron gene (SMN1). When the body produces insufficient SMN protein, motor neurons in the spinal cord degenerate and die, causing weakness and muscle shrinkage. Spinal muscular atrophy is the leading genetic cause of death in infants and toddlers. Built upon successful generation of spinal motor neurons from human embryonic stem cells in Dr. Li's laboratory, the project aims to better understand why motor neurons are affected in this disorder and establish a platform for testing therapies that could successfully treat this debilitating and fatal genetic disorder.


$450,000 – Targeting Lineage Committed Stem Cells to Damaged Intestinal Mucosa.

Daniel W. Rosenberg, Ph.D., UConn Health and Charles Giardina, Ph.D., UConn.

The intestinal mucosa, the innermost membranes of the intestinal wall, can be damaged in a variety of ways, including damage from inflammatory bowel disease and radiation therapy. Fortunately, these membranes also provide an excellent experimental system for studying tissue renewal and repair. The study explores new ways to induce human embryonic stem cells to become intestinal cells that can be used to repair damage.


$450,000 – Production and Validation of Patient-Matched Pluripotent Cells.

Theodore Rasmussen, Ph.D., UConn.

Regenerative medicine rests not just upon the capacity to generate and manage stem cell lines, but the ability to successfully transplant immunologically matched cells into patients. Because of this there is considerable interest in the nuclear reprogramming of somatic cells, the cells that make up the human body. This is a process to can revert a patient's own cells to an earlier development state, similar to stem cells. Like stem cells, those reprogrammed cells can be differentiated into many therapeutically-useful cell types. And since they contain the patient's own genome, they are immunologically matched to the patient. The research explores methods for inducing such cells, and assessing their usefulness and safety.


$450,000 – Tyrosine Phosphorylation Profiles Associated with Self-renewal and Differentiation of Human Embryonic Stem Cells.

Bruce Mayer, Ph.D., UConn Health.

The project aims to better understand the mechanics of how the switch between self-renewal and differentiation is controlled in human embryonic stem cells. Better understanding will make it possible for researchers to better control the behavior of cells in order to generate specialized cells that can be used to treat patients. One method of control is tyrosine phosphorylation, the addition of a phosphate group to the amino acid tyrosine in proteins. Dr. Mayer's group has recently developed a highly sensitive method to profile the entire spectrum of tyrosine phosphorylated proteins in a cell sample, enhancing the capacity to identify specific proteins that play a role in cell fate decisions.


$250,000 – Flow Cytometry Core for the Study of Human Embryonic Stem Cells at University of Connecticut.

Hector Leonardo Aguila, UConn Health.

Flow cytometry is a powerful microscopy technique used to identify rare cell types within complex populations of cells. It enables researchers to swiftly sort, count and examine microscopic particles, isolating cells for their homogeneity - or uniformity of structure and composition - so that their characteristics of cell division, cell death and metabolism can be evaluated. It's an essential tool for the University's stem cell program.


$200,000 – Differentiation of hESC Lines to Neural Crest Derived Trabecular Meshwork Like Cells - Implication in Glaucoma.

Dharamainder Choudhary, Ph.D., UConn Health.

Glaucoma is a major cause of blindness worldwide. A major risk factor is elevated pressure within the eye, which develops due to inadequate outflow of aqueous humor because cells in the eye's trabecular meshwork region have been damaged. Treatments generally involve either decreasing the rate at which aqueous humor, the watery substance filling the space between the lens and cornea, or surgically increasing the outflow. Neither treatment is ideal. The first goal of the project is to develop optimal conditions for differentiating human embryonic stem cells to a type with characteristics similar to trabecular meshwork cells.


$200,000 – Cytokine-induced Production of Transplantable Hematopoiectic Stem Cells from Human ES Cells.

Laijun Lai, Ph.D., UConn Health.

Hematopoietic stem cell transplantation (HCST), transplantation of stem cells from bone marrow, is the most common cell-based therapy applied today, used to treat a number of cancers, aplastic anemia, complications of irradiation and chemotherapy and many other diseases and conditions. HCST, especially in adults, is often limited by unavailability of freshly harvests stem cells from bone marrow, umbilical cord blood and mobilized peripheral blood. The first goal of the project is to evaluate alternative sources for cells for HCST.


$200,000 – Early Differentiation Markers in Human ES Cells: Identification and Characterization of Candidates.

Mark Carter, Principal Investigator, Center for Regenerative Biology, UConn.

Within a non-differentiating culture, stem cells are not identical with respect to marker gene expression. They may contain subpopulations of cells, and the differences are sometimes linked to differences in which tissues the cells ultimately generate. Because this heterogeneity may have consequences for stem cell function, the study focuses on transcription factor genes, genes that play a regulatory role in transfer of genetic information. Since proposed therapeutic uses of human ESCs rely on production of pure differentiated populations, it is essential to understand the variations within starting material, and how those differences impact efforts to direction cellular differentiation.