Disease Focus: Neurological Disorders
A hNSC Development Candidate for Huntington’s Disease
Huntington’s disease (HD) is a devastating degenerative brain disease with at least a 1 in 10,000 prevalence that inevitably leads to death. These numbers do not fully reflect the large societal and familial cost of HD, which requires extensive care-giving. HD has no effective treatment or cure and symptoms unstoppably progress for 15-20 years, with […]
Placental Stem Cells for the In Utero Treatment of Spina Bifida
Myelomeningocele – also known as spina bifida – is a devastating and costly defect that causes lifelong paralysis as well as bowel and bladder incontinence in newborns. It is one of the most common birth defects worldwide, with four children in the United States born with spina bifida every day. Spina bifida affects the physical, […]
Human Stem-Cell Based Development of a Potent Alzheimer’s Drug Candidate
Over 6 million people in the US suffer from Alzheimer’s disease (AD). There are no drugs that prevent the death of nerve cells in AD, nor has any drug been identified that can stimulate nerve cell replacement in aged human brain. Importantly, even if nerve cells could be replaced, the toxic environment of the AD […]
Injectable Hydrogels for the Delivery, Maturation, and Engraftment of Clinically Relevant Numbers of Human Induced Pluripotent Stem Cell-Derived Neural Progenitors to the Central Nervous System
One critical bottleneck in the translation of regenerative medicine into the clinic is the efficient delivery and engraftment of transplanted cells. While direct injection is the least invasive method for cell delivery, it commonly results in the survival of only 5-20% of cells. Studies suggest that delivery within a carrier gel may enhance cell viability, […]
Skin-derived precursor cells for the treatment of enteric neuromuscular dysfunction
The intestine performs the essential function of absorbing food and water into the body. Without a functional intestine, children and adults cannot eat normal meals, and these patients depend on intravenous nutrition to sustain life. Many of these patients do not have a neural system that coordinates the function of the intestine. These patients have […]
Skin-derived precursor cells for the treatment of enteric neuromuscular dysfunction
The intestine performs the essential function of absorbing food and water into the body. Without a functional intestine, children and adults cannot eat normal meals, and these patients depend on intravenous nutrition to sustain life. Many of these patients do not have a neural system that coordinates the function of the intestine. These patients have […]
Optimizing the differentiation and expansion of microglial progenitors from human pluripotent stem cells for the study and treatment of neurological disease.
Microglia are a type of immune cell within the brain that profoundly influence the development and progression of many neurological disorders. Microglia also inherently migrate toward areas of brain injury, making them excellent candidates for use in cell transplantation therapies. Despite the widely accepted importance of microglia in neurological disease, methods to produce microglia from […]
Engineered Biomaterials for Scalable Manufacturing and High Viability Implantation of hPSC-Derived Cells to Treat Neurodegenerative Disease
Cell replacement therapies (CRTs) have considerable promise for addressing unmet medical needs, including incurable neurodegerative diseases. However, several bottlenecks hinder CRTs, especially the needs for improved cell manufacturing processes and enhanced cell survival and integration after implantation. Engineering synthetic biomaterials that present biological signals to support cell expansion, differentiation, survival, and/or integration may help overcome […]
User-friendly predictive molecular diagnostic assays for quality control of stem cell derivatives for transplantation and drug discovery
Three years ago, with help from CIRM funding, we developed an assay. This is a genomics-base diagnostic assay, similar to those now used for diagnosing cancers; but in our case, it is designed to analyze human ES and iPS cells. The assay is very simple to use; researchers use microarrays to profile the genes that […]
Development of a Relevant Pre-Clinical Animal Model as a Tool to Evaluate Human Stem Cell-Derived Replacement Therapies for Motor Neuron Injuries and Degenerative Diseases
Motor neurons degenerate and die as a consequence of many conditions, including trauma to the spinal cord and its nerve roots and degenerative diseases such as amyotrophic lateral sclerosis and spinal muscular atrophy. Paralysis and in many cases death may result from a loss of motor neurons. No effective treatments are available for these patients. […]