Stage of Program: Translational Tool/Bottleneck
Antibody tools to deplete or isolate teratogenic, cardiac, and blood stem cells from hESCs
Purity is as important for cell-based therapies as it is for treatments based on small-molecule drugs or biologics. Pluripotent stem cells possess two properties: they are capable of self regeneration and they can differentiate to all different tissue types (i.e. muscle, brain, heart, etc.). Despite the promise of pluripotent stem cells as a tool for […]
Use of iPS cells (iPSCs) to develop novels tools for the treatment of spinal muscular atrophy.
Spinal Muscular Atrophy (SMA) is one of the most common lethal genetic diseases in children. One in thirty five people carry a mutation in a gene called survival of motor neurons 1 (SMN1) which is responsible for this disease. If two carriers have children together they have a one in four chance of having a […]
Engineering Defined and Scaleable Systems for Dopaminergic Neuron Differentiation of hPSCs
Human pluripotent stem cells (hPSC) have the capacity to differentiate into every cell in the adult body, and they are thus a highly promising source of differentiated cells for the investigation and treatment of numerous human diseases. For example, neurodegenerative disorders are an increasing healthcare problem that affect the lives of millions of Americans, and […]
Development of Single Cell MRI Technology using Genetically-Encoded Iron-Based Reporters
Clinical application of cell transplantation therapy requires a means of non-invasively monitoring these cells in the patient. Several imaging modalities, including MRI, bioluminescence imaging, and positron emission tomography have been used to track stem cells in vivo. For MR imaging, cells are pre-loaded with molecules or particles that substantially alter the image brightness; the most […]
Editing of Parkinson’s disease mutation in patient-derived iPSCs by zinc-finger nucleases
The goal of this proposal is to establish a novel research tool to explore the molecular basis of Parkinson’s disease (PD) – a critical step toward the development of new therapy. To date, a small handful of specific genes and associated mutations have been causally linked to the development of PD. However, how these mutations […]
Development and preclinical testing of new devices for cell transplantation to the brain.
The surgical tools currently available to transplant cells to the human brain are crude and underdeveloped. In current clinical trials, a syringe and needle device has been used to inject living cells into the brain. Because cells do not spread through the brain tissue after implantation, multiple brain penetrations (more than ten separate needle insertions […]
Robust generation of induced pluripotent stem cells by a potent set of engineered factors
The recent discovery of iPSC (induced Pluripotent Stem Cell) technology marks a promising breakthrough in regenerative medicine. The beauty of the technology is its ability to convert adult mature cells into embryonic stem cells through the expression of a cocktail of essential factor genes. Thus, iPSCs bypass the ethical dilemma of using embryonic materials and […]
Preparation and Delivery of Clinically Relevant Numbers of Stem Cells Using 3D Hydrogels
A critical bottleneck to translate the promise of regenerative medicine to the clinic is the ability to efficiently harvest, expand, and deliver sufficient numbers of viable stem cells. While relatively large numbers of patient-specific, multipotent human adipocyte stem cells (hASC) can be harvested from adults, these cells must be re-delivered to the patient (either with […]
Developing a method for rapid identification of high-quality disease specific hIPSC lines
Elucidating how genetic variation contributes to disease susceptibility and drug response requires human Induced Pluripotent Stem Cell (hIPSC) lines from many human patients. Yet, current methods of hIPSC generation are labor-intensive and expensive. Thus, a cost-effective, non-labor intensive set of methods for hIPSC generation and characterization is essential to bring the translational potential of hIPSC […]
Development of small molecule screens for autism using patient-derived iPS cells
Autism Spectrum Disorders (ASDs) are a heritable group of neuro-developmental disorders characterized by language impairments, difficulties in social integrations, and the presence of stereotyped and repetitive behaviors. There are no treatments for ASDs, and very few targets for drug development. Recent evidence suggests that some types of ASDs are caused by defects in calcium signaling […]