Stem Cell Therapy for Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is the most common and serious form of muscular dystrophy. One out of every 3500 boys is born with the disorder, and it is invariably fatal.…
Developing a therapeutic candidate for Canavan disease using induced pluripotent stem cell
Canavan disease is a devastating disease of infants which affects their neural development and leads to mental retardation and early death. It occurs in 1 in 6,400 persons in the…
New Drug Discovery for SMA using Patient-derived Induced Pluripotent Stem Cells
Spinal muscular atrophy (SMA) is the leading genetic cause of infant death in the U.S. This devastating disease affects 1 child in every 6,000-10,000 live births, with a North American…
Developing a drug-screening system for Autism Spectrum Disorders using human neurons
Autism and autism spectrum disorders (ASD) are complex neurodevelopmental diseases that affect 1 in 150 children in the United States. Such diseases are mainly characterized by deficits in verbal communication,…
Identification of hESC-mediated molecular mechanism that positively regulates the regenerative capacity of post-natal tissues
The tissue regenerative capacity deteriorates with age in animals and in humans, leading to the loss of organ function, which is well exemplified in skeletal muscle, but is poorly understood…
Engineered immune tolerance by Stem Cell-derived thymic regeneration
Stem cell therapies have the potential to transform medicine by allowing the regeneration of tissues or organs damaged by disease or trauma. In order for stem cell therapies to proceed,…
Maternal and Fetal Immune Responses to In Utero Hematopoietic Stem Cell Transplantation
The immune system is the body’s defense system against disease and can recognize foreign cells. Because of this, stem cells and organs that are transplanted from one person to another…
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…
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…
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…