Therapeutic/Technology: Therapeutic Approach
Identification of Novel Therapeutics for Danon Disease Using an iPS Model of the Disease
Autophagy is the cells mechanism for breaking down and recycling proteins. Danon disease is an inherited disorder of autophagy. Patients with this disease have major abnormalities in heart and skeletal muscle and generally die by the time they are in their 20s. Recently we used a new technology to turn skin cells from two patients […]
Extracellular Matrix Bioscaffold Augmented with Human Stem Cells for Cardiovascular Repair
An estimated 16.3 million Americans suffer from coronary heart disease. Every 25 seconds, someone has a coronary event and every minute, someone dies from one. Treatment for coronary heart disease has improved greatly in recent years, yet 1 in 6 deaths in the US in 2007 was still caused by this terrible disease. Stem cells […]
Autologous iPSC Therapy for Urinary Incontinence
Urinary incontinence (UI) is common and serious, with two-thirds of the burden borne by women. UI impacts both quality and length of life; women with UI suffer debilitating falls, experience social isolation and are clinically-depressed more commonly than continent women. UI is the primary reason for elderly women to be institutionalized and carries an annual […]
Functional Neural Relay Formation by Human Neural Stem Cell Grafting in Spinal Cord Injury
We aim to develop a novel stem cell treatment for spinal cord injury (SCI) that is substantially more potent than previous stem cell treatments. By combining grafts of neural stem cells with scaffolds placed in injury sites, we have been able to optimize graft survival and filling of the injury site. Grafted cells extend long […]
Tissue engineered cartilage from autologous, dermis-isolated, adult, stem (DIAS) cells
This study addresses the cartilage defects resulting from injuries or from wear-and-tear that can eventually degenerate to osteoarthritis. This is a significant problem that impacts millions and costs in excess of $65B per annum in the US alone. Addressing this indication successfully holds potential for halting the progression of cartilage damage before it destroys the […]
Multiple Sclerosis therapy: Human Pluripotent Stem Cell-Derived Neural Progenitor Cells
Multiple Sclerosis (MS) is a disease of the central nervous system (CNS) caused by inflammation and loss of cells that produce myelin, which normally insulates and protects nerve cells. MS is a leading cause of neurological disability among young adults in North America. Current treatments for MS include drugs such as interferons and corticosteroids that […]
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 […]
Human ES cell-derived MGE inhibitory interneuron transplantation for spinal cord injury
Transplantation of neuronal precursors into the central nervous system offers great promise for the treatment of neurological disorders including spinal cord injury (SCI). Among the most significant consequences of SCI are bladder spasticity and neuropathic pain, both of which likely result from a reduction in those spinal inhibitory mechanisms that are essential for normal bladder […]
Molecules to Correct Aberrant RNA Signature in Human Diseased Neurons
Approximately 5,600 people in the U.S. are diagnosed with ALS each year. The incidence of ALS is two per 100,000 people, and it is estimated that as many as 30,000 Americans may have the disease at any given time. There are no effective therapies of ALS to-date. Recent genetic discoveries have pinpointed mutations that lead […]
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. […]