Stage of Program: Preclinical Research
Targeting Stem Cells to Enhance Remyelination in the Treatment of Multiple Sclerosis
Multiple sclerosis (MS) is an autoimmune disease in which the myelin sheath that insulates neurons is destroyed, resulting in loss of proper neuronal function. Existing treatments for MS are based on strategies that suppress the immune response. While these drugs do provide benefit by reducing relapses and delaying progression (but have significant side effects), the […]
Regeneration of Functional Human Corneal Epithelial Progenitor Cells
Over 3.2 million people worldwide are bilateral blind from corneal diseases. Limbal stem cell deficiency (LSCD) has been recognized as a major cause, either primary or secondary, of significant visual loss and blindness in many common corneal disorders. A healthy, transparent ocular surface is made up of non-keratinized, stratified squamous epithelium that is highly differentiated. […]
Liver Cell Transplantation
Because there is still considerable morbidity and mortality associated with the process of whole liver transplantation, and because more than a thousand people die each year while on the liver transplantation list, and tens of thousands more never get on the list because of the lack of available livers, it is evident that improved and […]
Development of RNA-based approaches to stem cell gene therapy for HIV
Despite significant advances in treatment and prevention programs, HIV infection with progression to Acquired Immunodeficiency Syndrome (AIDS) is still prevalent in California. The CDC Estimates >56,000 new cases of HIV infection each year in the US with over 148,000 cumulative cases reported in California alone (as of 2009). Multi-drug therapy has been helpful in reducing […]
Stem cell-based carriers for RCR vector delivery to glioblastoma
Modified viruses can be used to infect tumor cells and alter the tumor cell to make anti-tumor proteins. Most researchers use virus that can infect and modify the tumor cell it enters, but can not make more of itself to infect additional cells surrounding the original infected cell. This type of virus is called replication-incompetent […]
Human retinal progenitor cells as candidate therapy for retinitis pigmentosa
The targeted disease is retinitis pigmentosa (RP), is a severe form of blindness that runs in families. This disease is not overly common, yet represents an attainable near term target for stem cell therapy for a number of reasons: 1) RP destroys the light detecting cells of the retina but generally leaves the rest of […]
Dual targeting of tyrosine kinase and BCL6 signaling for leukemia stem cell eradication
Leukemia is the most frequent form of cancer in children and teenagers, but is also common in adults. Chemotherapy has vastly improved the outcome of leukemia over the past four decades. However, many patients still die because of recurrence of the disease and development of drug-resistance in leukemia cells. In preliminary studies for this proposal […]
Dual targeting of tyrosine kinase and BCL6 signaling for leukemia stem cell eradication
Leukemia is the most frequent form of cancer in children and teenagers, but is also common in adults. Chemotherapy has vastly improved the outcome of leukemia over the past four decades. However, many patients still die because of recurrence of the disease and development of drug-resistance in leukemia cells. In preliminary studies for this proposal […]
Banking transplant ready dopaminergic neurons using a scalable process
Parkinson’s disease (PD) is a devastating movement disorder caused by the death of dopaminergic neurons (a type of nerve cells in the central nervous system) present in the midbrain. These neurons secrete dopamine (a signaling molecule) and are a critical component of the motor circuit that ensures movements are smooth and coordinated. All current treatments […]
A hESc-based Development Candidate for Huntington’s Disease
Huntington’s disease (HD) is a devastating degenerative brain disease with 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 caregiving. HD has no effective treatment or cure and symptoms unstoppably progress for 15-20 years, with onset typically […]