Project Objective: Tool/Resources/Bottleneck
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 […]
Use of hiPSCs to develop lead compounds for the treatment of genetic diseases
This study will use Ataxia-Telangiectasia (A-T), an early-onset inherited neurodegenerative disease of children, as a model to study the mechanisms leading to cerebellar neurodegeneration and to develop a drug that can slow or halt neurodegeneration. We will start with skin cells that were originally grown from biopsies of patients with A-T who specifically carry “nonsense” […]
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 […]
Magnetic Particle Imaging: A Novel Ultra-sensitive Imaging Scanner for Tracking Stem Cells In Vivo
We aim to develop, test and validate a new, sensitive and affordable scanner for tracking the location of injected cells in humans and animals. This new scanning method, called Magnetic Particle Imaging, will ultimately be used to track the location and viability of stem cells within the human body. It could solve one of the […]
Development of Synthetic Microenvironments for Stem Cell Growth and Differentiation
Currently, many chronic diseases and injuries do not have effective cures; millions of people suffer from disabilities while carrying on daily lives without appropriate medical assistance. Advances in human pluripotent stem cells (hPSCs) research have provided the potential hope for significant improvements of disease treatment and management. The success of stem cell-based therapy will have […]
Development of a Hydrogel Matrix for Stem Cell Growth and Neural Repair after Stroke
Stroke is the leading cause of adult disability. Most patients survive their initial stroke, but do not recover fully. Because of incomplete recovery, up to 1/3 of stroke patients are taken from independence to a nursing home or assisted living environment, and most are left with some disability in strength or control of the arms […]
Site-specific integration of Lmx1a, FoxA2, & Otx2 to optimize dopaminergic differentiation
The objective of this study is to develop a new, optimized technology to obtain a homogenous population of midbrain dopaminergic (mDA) neurons in a culture dish through neuronal differentiation. Dopaminergic neurons of the midbrain are the main source of dopamine in the mammalian central nervous system. Their loss is associated with one of the most […]
Development and Application of Versatile, Automated, Microfluidic Cell Culture System
Supported in part by a previous CIRM Tools and Technologies Grant [REDACTED], we have optimized and scaled up highly advanced (microfluidic) cell culture chips into manufacturable form, produced prototype instruments to drive these chips, and demonstrated that we can culture cells, dose them with combinations of reagents, and export them back off the chip. Since […]
Pluripotent and Somatic Stem Cell Models to Study Inherited Diarrheal Disorders
Our research group at [REDACTED] has had a long-standing interest in understanding the cause of several disorders that result in severe, and often times fatal forms of diarrhea in children. These diarrheal disorders are inherited, and somehow lead to poor absorption of nearly all forms of nutrients, including protein, sugars and fats. Why children with […]
Single Molecule Biophysics and Biology of Cellular Identity
One of your earliest childhood biology lessons probably occurred when your body demonstrated to you that your skin is an organ that is able to self-regenerate. Indeed wound healing is a fascinating process in which cells carry out a precise and complex choreography that includes cellular differentiation and regulation of gene expression. Our lab studies […]