Disease Focus: Skeletal/Smooth Muscle disorders
Combination therapy to Enhance Antisense Mediated Exon Skipping for Duchenne Muscular Dystrophy
A drug was identified through the use of muscle stem cells that can enhance the effectiveness of exon skipping by antisense oligonucleotides to the DMD gene to restore dystrophin expression and at least partially correct the defect responsible for loss of muscle function in Duchenne. We propose to test the effectiveness of this drug in […]
Phenotypic Analysis of Human ES Cell-Derived Muscle Stem Cells
We study human muscle development, and are actively investigating potential cell-based therapies for the treatment of degenerative muscle diseases, such as muscle dystrophy. This project will define the pathway that muscle stem cells follow as they form new muscle, and identify which muscle stem cells are most useful for therapy. Our approach will be to […]
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. Until recently, there was little hope that the widespread muscle degeneration that accompanies this disease could be combated. However, stem cell therapy now offers that […]
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 in molecular terms. Our recent work uncovered that factors produced by human embryonic stem cells have a unique ability to enhance the regenerative responses of […]
Skeletal muscle development from hESC and its in vivo applications in animal models of muscular dystrophy
Embryonic stem cells (ESC) originating from early stage embryos are able to differentiate into any type of cells in the body. The generation of ESC lines from human embryos (hESC) has attracted a lot of dispute among researchers, but raised the hope that one day hESCs can be used in cell replacement therapy for the […]
Purified allogeneic hematopoietic stem cells as a platform for tolerance induction
Blood and immune cells originate and mature in the bone marrow. Bone marrow cells are mixtures of blood cells at different stages of development, and include rare populations of blood-forming stem cells. These stem cells are the only cells capable of generating the blood system for the life of an individual. Bone marrow transplants (BMT) […]
Regulation of Stem Cell Fate in Bioengineered Arrays of Hydrogel Microwells
Stem cell biology, since its inception 30 years ago, has been hindered by our limited ability to observe and direct the decisions of individual stem cells. In the case of adult tissue-specific stem cells, such as those from blood, muscle or pancreas, the numbers available for clinical use are extremely limited, as in tissue culture […]
A Novel Microenvironment-Mediated Functional Skeletal Muscle from Human Embryonic Stem Cells and their In Vivo Engraftment
Muscle wasting is a serious clinical problem associated with a number of diseases and health conditions, affecting individuals of all ages. Muscular dystrophy (MD) is a form of muscle wasting disease resulting from genetic mutations. Duchenne muscular dystrophy (DMD) is the most common form of MD that limits motility and life expectancy of children. It […]
Generation of clinical grade human iPS cells
The therapeutic use of stem cells depends on the availability of pluripotent cells that are not limited by technical, ethical or immunological considerations. The goal of this proposal is to develop and bank safe and well-characterized patient-specific pluripotent stem cell lines that can be used to study and potentially ameliorate human diseases. Several groups, including […]
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 […]