Stage of Program: Preclinical Research

Retinal progenitor cells for treatment of retinitis pigmentosa

Retinitis pigmentosa (RP) is a severe form of blindness that runs in families with an incidence of 1:4000. A team at UC Irvine, is using cells called retinal progenitor cells to repair the damage caused by this vision destroying disease. The cells are injected into the back of the eye and it’s hoped they will […]

Placental Derived Natural Killer Cells to Target Solid Tumor Cancer Stem Cells (CSC)

Translational Candidate Placental-derived stem cells becoming a unique NK-like cell Area of Impact solid tissue cancers Mechanism of Action These expanded cells share many characteristics as NK cells but appear to represent a unique stage in differentiation and a cell-type with advantageous properties. These cells have been demonstrated to persist for much longer periods of […]

Development of a humanized mouse model for testing anti-HIV HSPC gene therapy strategies in HIV-1 infected mice.

Combination therapy to Enhance Antisense Mediated Exon Skipping for Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) affects 1 in every 3,500 boys worldwide. DMD is caused by mutations in the gene encoding dystrophin, a protein key to muscle health. DMD patients are typically weaker than normal by age 3, and with progressive muscle weakness most loose the ability to walk by age 11. DMD progresses to complete […]

Clinical Development of an N-cadherin Antibody to Target Cancer Stem Cells

Metastatic disease and the castration resistance remain tremendous challenges in the treatment of prostate cancer. New targeted treatments, such as the ant-testosterone medication enzalutamide, have improved the survival of men with advanced disease, but a majority develops treatment resistance. The field of cancer stem cells hypothesizes that treatment resistance emerges because stem cells are inherently […]

Use of human iPSC-derived neurons from Huntington’s Disease patients to develop novel, disease-modifying small molecule structural corrector drug candidates targeting the unique, neurotoxic conformation of mutant huntingtin

The long-term objective of this project is to develop a drug to treat Huntington’s disease (HD), the most common inherited neurodegenerative disorder. Characterized by involuntary movements, personality changes and dementia, HD is a devastatingly progressive disease that results in death 10–20 years after disease onset and diagnosis. No therapy presently exists for HD; therefore, this […]

Programming Human ESC-derived Neural Stem Cells with MEF2C for Transplantation in Stroke

The goal of this project is to produce a stem cell-based therapy for stroke (also known as an ischemic cerebral infarct). Stroke is the third leading cause of death in the USA, and a leading cause of disability among adults. Currently, there are no effective treatments once a stroke has occurred (termed completed stroke). In […]

Programming Human ESC-derived Neural Stem Cells with MEF2C for Transplantation in Stroke

Gene Targeting to Endogenous Stem Cells for Segmental Bone Fracture Healing

Segmental bone fractures are a complex medical condition. These injuries cause great suffering to patients, long-term hospitalization, repeated surgeries, loss of working days, and considerable costs to the health system. It is well known that bone grafts taken from the patient (autografts) are considered the gold-standard therapy for these bone defects. Yet these grafts are […]

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. Life expectancy of ALS patients averages 2–5 years from diagnosis. About 5,600 people in the U.S. are diagnosed with ALS each year, and about 30,000 […]

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