Award Type: Development Candidate Feasibility Award
Stem cell based small molecule therapy for Alzheimer’s disease
Over 6 million people in the US suffer from AD. There are no drugs that prevent the death of nerve cells in AD, nor has any drug been identified that can stimulate their replacement. Even if nerve cells could be replaced, the toxic environment of the brain will kill them unless they are protected by […]
Identifying Drugs for Alzheimer’s Disease with Human Neurons Made From Human IPS cells
We propose to discover new drug candidates for Alzheimer’s Disease (AD), which is common, fatal, and for which no effective disease-modifying drugs are available. Because no effective AD treatment is available or imminent, we propose to discover novel candidates by screening purified human brain cells made from human reprogrammed stem cells (human IPS cells or […]
Human ES cell based therapy of heart failure without allogenic immune rejection
Heart failure is a major and ever-growing health problem affecting an estimated 5.8 million Americans with about half a million new cases every year. There are limited therapeutic options for heart failure. Heart transplantation is effective but has limited impact due to scarcity of donor organs and eventual immune rejection even under chronic immune suppression. […]
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 […]
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 […]
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 […]
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 […]