Engineered iPSC for therapy of limb girdle muscular dystrophy type 2B
Limb girdle muscular dystrophy type 2B (LGMD 2B) is a form of muscular dystrophy that leads to muscle degeneration and disability. In LGMD 2B, a vital muscle protein is mutated,…
Development of Novel Autophagy Inducers to Block the Progression of and Treat Amyotrophic Lateral Sclerosis (ALS) and Other Neurodegenerative Diseases
ALS is a progressive neurodegenerative disease that primarily affects motor neurons (MNs). It results in paralysis and loss of control of vital functions, such as breathing, leading to premature death.…
Human iPSC modeling and therapeutics for degenerative peripheral nerve disease
The applicant is an MD/PhD trained physician scientist, whose clinical expertise is neuromuscular disorders including peripheral nerve disease. The proposal is aimed at providing a research proposal and career development…
Stem cell therapy for inflammatory bowel disease
One of the most promising approaches that physicians foresee for treating human disease is regenerative medicine. A major aim in this field is to restore function by repairing damaged organs.…
White matter neuroregeneration after chemotherapy: stem cell therapy for “chemobrain”
Chemotherapy for cancer is often life saving, but it also causes a debilitating syndrome of impaired cognition characterized by deficits in attention, concentration, information processing speed, multitasking and memory. As…
Characterization of Human Skeletal Muscle Stem Cells for Clinical Application
Skeletal muscle makes up 40% of our bodies, dictates our form, is responsible for our ability to move, express ourselves, eat, breath and to look around. Restoration or preservation of…
Tunable hydrogels for therapeutic delivery of multipotent stem cells.
Rationale: Skin ulcers represent the largest economic burden of all skin diseases. Human adult stem cell therapies for the treatment of chronic wounds have shown considerable promise. However, a delivery…
Human Induced Pluripotent Stem Cell-Derived Cardiovascular Progenitor Cells for Cardiac Cell Therapy.
Despite therapeutic advances, cardiovascular disease remains a leading cause of mortality and morbidity in California. Regenerative therapies that restore normal function after a heart attack would have an enormous societal…
The generation and expansion of tissue-engineered small intestine from human stem/ progenitor cells: a preclinical study of functional translation
This proposal aims to complete the preclinical steps to develop tissue-engineered intestine (TESI) as a functional replacement of the small intestine to treat short bowel syndrome (SBS). Common birth conditions…
Human pluripotent stem cell-based therapeutics for preeclampsia
Preeclampsia (PE) is a pregnancy complication, characterized by high blood pressure and abnormal kidney function, which affects 5-8% of all pregnancies. It is responsible for a significant proportion of maternal…
