Disease Focus: Pediatrics

Stem cell differentiation to thymic epithelium for inducing tolerance to stem cells

The thymus is an organ that plays a key role in controlling immune responses and immune tolerance. The thymus promotes immune tolerance by deleting and removing self-reactive T cells from the immune system. In addition, the thymus also helps drive the production of important suppressor T cell populations like regulatory T cells that also control […]

Mechanism of Tissue Engineered Small Intestine Formation

Short Bowel Syndrome is an expensive, morbid condition with an increasing incidence. Fundamental congenital and perinatal conditions such as gastroschisis, malrotation, atresia, and necrotizing enterocolitis (NEC) may lead to short bowel syndrome (SBS). NEC is the most common gastrointestinal emergency in neonates and primarily occurs in premature infants As rates of prematurity are increasing, so […]

Development of Induced Pluripotent Stem Cells for Modeling Human Disease

Human embryonic stem cells (hESC) hold great promise in regenerative medicine and cell replacement therapies because of their unique ability to self-renew and their developmental potential to form all cell lineages in the body. Traditional techniques for generating hESC rely on surplus IVF embryos and are incompatible with the generation of genetically diverse, patient or […]

Generation of Pluripotent Cell Lines from Human Embryos

Human embryonic stem cells (hESCs) hold significant promise for regenerative medicine. In this application our goal is to derive hESC lines from pre-implantation embryos to generate a source of low passage lines that can be used in research and to develop the procedures required to generate a clinic grade cell-based product. In this application we […]

Functional Genomic Analysis of Chemically Defined Human Embryonic Stem Cells

Regenerative medicine holds the promise that tissues can be engineered in vitro and then transplanted into patients to treat debilitating diseases. Human Embryonic Stem Cells differentiate into a wide array of adult tissue types and are thought to be the best hope for future regenerative therapies. This grant has three main goals: 1. The creation […]

hESC as tools to investigate the neural crest origin of Ewing’s sarcoma

Human embryonic stem cells (hESC) hold great promise as sources of tissue for regenerative medicine and therapeutics. In addition, their utility as tools to study the origins and biology of human disease must not be underestimated. hESC give rise to tissue-specific adult stem cells and, ultimately, to all mature tissues in the body. As such, […]

Differentiation of Human Embryonic Stem Cells to Intestinal Fates

The roughly 25 feet of intestine in the adult human play numerous essential roles in daily life, such as nutrient absorption, secretion of hormones, and serving as a barrier to infection. Commensurate with these diverse roles, diseases of the intestine are a considerable source of human morbidity and mortality. Indeed, numerous pathologic conditions including inflammatory […]

Differentiation of Human Embryonic Stem Cells into Urothelium

Augmentation or replacement of the bladder is often necessary for the treatment of adults with bladder cancer and children with spinal cord injury or spina bifida. Current surgical techniques utilize segments of intestine or stomach as a substitute for bladder wall. Use of intestinal segments is associated with many complications including infection, stones, salt imbalance, […]

Self-renewal and senescence in iPS cells derived from patients with a stem cell disease

The discovery of induced pluripotent stem (iPS) cell technology promises to revolutionize our understanding of human disease and to allow the development of new cellular therapies for regenerative medicine applications. The ability to reprogram a patient’s fibroblasts to iPS cells creates the opportunity to expand human cells with a specific genetic defect and to study […]

Stem Cell Gene Therapy for Sickle Cell Disease

Sickle cell disease (SCD), which results from an inherited mutation in the hemoglobin gene that causes red blood cells to “sickle” under conditions of low oxygen, occurs with a frequency of 1/500 African-Americans, and is also common in Hispanic-Americans, who comprise up to 5% of SCD patients in California. The median survival based on 1991 […]