Disease Focus: Skeletal/Smooth Muscle disorders


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 […]

Derivation and characterization of human ES cells from FSHD embryos

Facioscapulohumeral muscular dystrophy (FSHD) is the third most common hereditary muscular dystrophy. It is autosomal dominant, meaning that if one of the parents has the disease, their children have a 50:50 chance of getting it, too. FSHD is characterized by progressive weakness and atrophy of facial, shoulder and upper arm musculature, which can spread to […]

Using human embryonic stem cells to treat radiation-induced stem cell loss: Benefits vs cancer risk

A variety of stem cells exist in humans throughout life and maintain their ability to divide and change into multiple cell types. Different types of adult derived stem cells occur throughout the body, and reside within specific tissues that serve as a reserve pool of cells that can replenish other cells lost due to aging, […]

A modular automation approach to stem cell modeling to increase throughput, reproducibility and access

This project enhances stem cell access, scalability, and collaboration. It offers characterized hPSC lines, CRISPR editing, and differentiation on automated platforms accelerating progress in biology, disease research, and regenerative medicine. The project benefits California by advancing regenerative medicine through diverse hPSC lines, potentially leading to novel treatments and addressing health disparities. It offers educational opportunities […]