Therapeutic/Technology: Therapeutic Approach


Direct Cardiac Reprogramming for Heart Regeneration

Heart disease is a leading cause of mortality. The underlying pathology is typically loss of heart muscle cells that leads to heart failure. Because heart muscle has little or no regenerative capacity after birth, current therapeutic approaches are limited for the over 5 million Americans who suffer from heart failure. Our recent findings regarding direct […]

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. […]

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 […]

Gene Correction of Autologous Hematopoietic Stem Cells in Artemis Deficient SCID

Artemis is a chemical in all cells in the body that is essential for the normal development of the immune system and repairing damaged DNA. Artemis deficiency (AD) causes Severe Combined Immunodeficiency (SCID-A), a “bubble baby” syndrome associated with increased sensitivity to radiation and chemotherapy. SCID-A is hard to treat with a bone marrow stem […]

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 […]