Stage of Program: Candidate Discovery (DISC2, some 1.0 projects)
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 […]
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 […]
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 […]
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 […]
Use of hiPSCs to develop lead compounds for the treatment of genetic diseases
This study will use Ataxia-Telangiectasia (A-T), an early-onset inherited neurodegenerative disease of children, as a model to study the mechanisms leading to cerebellar neurodegeneration and to develop a drug that can slow or halt neurodegeneration. We will start with skin cells that were originally grown from biopsies of patients with A-T who specifically carry “nonsense” […]
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. […]
Inhibitory Nerve Cell Precursors: Dosing, Safety and Efficacy
Many neurological disorders are characterized by an imbalance between excitation and inhibition. Our ultimate goal: to develop a cell-based therapy to modulate aberrant brain activity in the treatment of these disorders. Our initial focus is on epilepsy. In 20-30% of these patients, seizures are unresponsive to drugs, requiring invasive surgical resection of brain regions with […]
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. Until recently, there was little hope that the widespread muscle degeneration that accompanies this disease could be combated. However, stem cell therapy now offers that […]