Stem Cells Secreting GDNF for the Treatment of ALS
This project aims to use a powerful combined stem cell and gene therapy approach to treat patients with amyotrophic lateral sclerosis (ALS or Lou Gehrig’s Disease). ALS is a devastating…
Recombinant Bispecific Antibody Targeting Cancer Stem Cells for the Therapy of Glioblastoma
Glioblastoma multiforme is the most prevalent and aggressive type of brain tumor, and devastating to any patient unfortunate enough to receive its diagnosis. As the most populous state in the…
A monoclonal antibody that depletes blood stem cells and enables chemotherapy free transplants
Successful stem cell therapy requires replacement of diseased or dysfunctional stem cells with healthy ones. These healthy stem cells can come from either a donor or can be stem cells…
Anti-Notch1, OMP-52M51: A New Cancer Therapeutic to Reduce CSC Frequency
A important benefit of the tremendous progress in stem cell research has been the recognition that stem cell pathways are frequently re-activated in cancer cells conferring stem cell-like properties on…
Genetically Engineered Mesenchymal Stem Cells for the Treatment of Vertebral Compression Fractures.
Osteoporosis is an unsolved and highly prevalent health care problem: 10 million Americans suffer from the disease, and an additional 34 million have low bone mass. Roughly half of all…
Neuroprotection to treat Alzheimer’s: a new paradigm using human central nervous system cells
Alzheimer’s disease (AD) is an incurable disorder that affects memory, social interaction and the ability to perform everyday activities. In the USA alone, the number of AD patients aged 65…
Combination therapy to Enhance Antisense Mediated Exon Skipping for Duchenne Muscular Dystrophy
A drug was identified through the use of muscle stem cells that can enhance the effectiveness of exon skipping by antisense oligonucleotides to the DMD gene to restore dystrophin expression…
Preclinical Development and First-In-Human Testing of GRNCM1 in Advanced Heart Failure
This application seeks to bring to the clinic a new treatment for myocardial disease based on human embryonic stem cell (hESC) derived cardiomyocytes. hESC-cardiomyocytes have the unique potential to address…
Neural Stem Cell-Based Therapy For Parkinson’s Disease
Ongoing degeneration of dopaminergic (DA) neurons in the midbrain is the hallmark of Parkinson’s disease (PD), a movement disorder that manifests with tremor, bradykinesia and rigidity. One million Americans live…
hESC-derived NPCs Programmed with MEF2C for Cell Transplantation in Parkinson’s Disease
We proposes to use human embryonic stem cells (hESCs) differentiated into neural progenitor/stem cells (NPCs), but modified by transiently programming the cells with the transcription factor MEF2C to drive them…