Project Objective: Research Insights

Mechanism and Utility of Direct Neuronal Conversion with a MicroRNA-Chromatin Switch

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 to become functioning neurons. A number of barriers must be traversed to bring these goals to practical therapies. Recently our laboratory and others have found […]

Asymmetric stem cell division oriented by a local self-renewing signal

When stem cells divide, the two daughter cells have a choice. Commonly, one of the daughter cells becomes a new stem cell while the other one will be more specialized (or differentiated). This property — the ability to generate more stem cells (self-renewal) while making differentiated cells simultaneously — defines a stem cell. Stem cells […]

3D Modeling of Retina using Polymer Scaffolds for Understanding Disease Pathogenesis

Inherited retinal degenerations result in visual loss in patients as early as in their adolescence. Retinitis Pigmentosa includes a group of such degenerations which run in families and can result in legal blindness by 40 years of age. Even though we know by now a number of gene mutations which can cause these disorders, we […]

Modeling disease in human embryonic stem cells using new genetic tools

The use of stem cells or stem cell-derived cells to treat disease is one important goal of stem cell research. A second, important use for stem cells is the creation of cellular models of human development and disease, critical for uncovering the molecular roots of illness and testing new drugs. However, a major limitation in […]

Investigation of the role of O-GlcNAcylation in SOX2 function during reprogramming

Induced pluripotent stem cells (iPSCs) are a potential source of material for cell replacement therapies. Thus, achieving maximal efficiency of reprogramming will be important for cellular medicine. In this submission we propose to test whether manipulating the mammalian glucose sensing pathway. The enzyme OGT is part of the glucose-sensing pathway and is necessary in pluripotent […]

Induction of Pluripotent Stem Cell-Derived Pacemaking Cells

Currently, over 350,000 patients per year with abnormal heart rhythm receive electronic pacemakers to restore their normal heart beat. Electronic pacemakers do not respond to the need for changing heart rate in situations such as exercise and have limited battery life, which can be resolved with biopacemakers. In this proposed project, we will examine methods […]

Long noncoding RNAs for pluripotency and cell fate commitment

The human body is composed of thousands of cell types, which all came originally from embryonic stem cells. Although all these cell types have the same genetic blueprint, different genes are active in different cells to give each its distinctiveness. The process by which the genes remember whether they are in liver, brain, or skin […]

Role of the NMD RNA Decay Pathway in Maintaining the Stem-Like State

A subset of intellectual disability cases in humans are caused by mutations in an X-linked gene essential for a quality control mechanism called nonsense-mediated RNA decay (NMD). Patients with mutations in this gene—UPF3B—commonly have not only ID, but also schizophrenia, autism, and attention-deficit/hyperactivity disorder. Thus, the study of UPF3B and NMD may provide insight into […]

Studying Arrhythmogenic Right Ventricular Dysplasia with patient-specific iPS cells

Most heart conditions leading to sudden death or impaired pumping heart functions in the young people (

Mechanisms to protect hESC-derived cells from allogenic immune rejection

The potential of human embryonic stem cells (hESC) to differentiate into a tremendous range of biologically active cells/tissues is the basis for many novel therapeutic strategies. However, immune-mediated rejection of hESC-derived tissues by the patient remains a significant barrier to the promise of regenerative therapies. Therefore, it is key to develop strategies to induce immunological […]