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.…
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…
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)…
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…
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…
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…
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…
Interrogating Satellite Cell and Myofiber Defects and Repair in Human DMD using Single Nuclei/Single Cell RNA Sequencing of Muscle Resident Cells
Research Objective We will describe, for the first time, human muscle satellite cell, myofiber and immune cell dynamics due to dystrophin deficiency and AAV gene therapy in human muscle at…
Engineering AAV capsids for transduction of neural and muscle stem cells
Research Objective The studies will identify and characterize new gene therapy vectors able to deliver gene editing components to stem cells. to enable treatment of diseases involving both muscle and…
Caleb’s Courage: Stem Cell Therapy for Duchenne Muscular Dystrophy
At its July meeting, the CIRM governing Board was honored to hear from Caleb Sizemore, a delightful and courageous young man who was born with Duchenne muscular dystrophy, a fatal…
