Human Stem Cell Use: Embryonic Stem Cell
In Utero Embryonic Stem Cell Transplantation to Treat Congenital Anomalies
Many fetuses with congenital blood stem cell disorders such as sickle cell disease or thalassemia are prenatally diagnosed early enough in pregnancy to be treated with stem cell transplantation. The main benefit to treating these diseases before birth is that the immature fetal immune system may accept transplanted cells without needing to use immunosuppressant drugs […]
Stem cell therapy for inflammatory bowel disease
One of the most promising approaches that physicians foresee for treating human disease is regenerative medicine. A major aim in this field is to restore function by repairing damaged organs. Inflammatory bowel disease (IBD) is a chronic disease characterized by intermittent episodes of intestinal inflammation and disruption of the intestinal epithelial barrier. It causes significant […]
Preclinical evaluation of human embryonic stem cell-derived cardiovascular progenitors in a large animal model
Because the regenerative capacity of adult heart is limited, any substantial cell loss as a result of a heart attack is mostly irreversible and may lead to progressive heart failure. Human pluripotent stem cells can be differentiated to heart cells, but their properties when transplanted into an injured heart remain unresolved. We propose to perform […]
Epigenetic regulation of human cardiac differentiation
Each cell type in our body has its own identity. This identity allows a heart cell to contract repetitively, and a brain cell to conduct nerve impulses. Each cell type gains its identity by turning on or off thousands of genes that together give the cell its identity. Understanding how these sets of genes are […]
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 […]
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 […]
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 […]