Stage of Program: Candidate Discovery (DISC2, some 1.0 projects)
Heart Repair with Human Tissue Engineered Myocardium
Heart disease is the number one cause of morbidity and mortality in the US. With an estimated 1.5 million new or recurrent myocardial infarctions, the total economic burden on our health care system is enormous. Although conventional pharmacotherapy and surgical interventions often improve cardiac function and quality of life, many patients continue to develop refractory […]
Local Delivery of Rejuvenated Old Muscle Stem Cells to Increase Strength in Aged Patients
As humans age, the ability to regenerate skeletal muscle tissue is impaired. Injuries to the musculoskeletal system that require extended periods of immobilization lead to muscle atrophy and are particularly devastating to the elderly population. Loss of skeletal muscle mass and function reduces mobility, which negatively affects quality of life, and increases the risk of […]
Generation of hepatic cell from placental stem cell for congenital metabolic disorders
Approximately 1 in 1,500 children has a congenital metabolic disorder. These inborn errors of metabolism are caused by deficiencies of different enzymes and result in accumulation of various substances inside cells. These substances affect the function of vital organs, and in many cases are lethal. Transplantation of cells that possess the particular deficient enzyme carries […]
Immune-Matched Neural Stem Cell Transplantation for Pediatric Neurodegenerative Disease
Children with inherited degenerative diseases of the brain will be among the first to benefit from novel approaches based on stem cell therapy (SCT). This assertion is based on a number of medical and experimental observations and precedents including: 1) These diseases currently lack effective therapies and can cause profound mental retardation or lead to […]
Generation of safe and therapeutically effective human induced hepatocyte-like cells
Although the liver can regenerate itself, chronic or overwhelming damage can cause life-threatening liver failure. Currently, the only therapy for liver failure is liver transplantation. Because the supply of cadaveric livers or liver tissue from living donors far exceeds the demand, physicians and researchers seek to develop new therapies to save the lives of patients […]
Direct reprogramming towards vascular progenitors for the treatment of ischemia
Angiogenesis or the generation of new blood vessels is a critical part of the normal healing process. Newly created vessels ensure the delivery of oxygen, nutrients, and specific repair signals to injured tissues. Indeed, even though additional repair mechanisms are required, such as replenishment of tissue-specific cell types, angiogenesis contributes to the healing of a […]
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 […]