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…
Neural and general splicing factors control self-renewal, neural survival and differentiation
Human embryonic and patient-specific induced pluripotent stem cells have the remarkable capacity to differentiate into many cell-types, including neurons, thus enabling the modeling of human neurological diseases in vitro, and…
Molecular Imaging for Stem Cell Science and Clinical Application
Stem cells offer tremendous potential to treat previously intractable diseases. The clinical translation of these therapies, however, presents unique challenges. One challenge is the absence of robust methods to monitor…
Molecular mechanisms of neural stem cell differentiation in the developing brain
One of the most exciting possibilities in stem cell biology is the potential to replace damaged or diseased neural tissues affected by neurodegenerative disorders. Stem-cell-derived neurons provide a potentially limitless…
Generation of disease models for neurodegenerative disorders in hESCs by gene targeting
The ability to target a specific locus in the mouse genome and to alter it in a specific fashion has fundamentally changed experimental design and made mice the preeminent model…
High throughput modeling of human neurodegenerative diseases in embryonic stem cells
An important class of neurological diseases predominantly affects spinal motor neurons, the neurons that control muscle movement. The most well known of these motor neuronopathies is Amyotrophic Lateral Sclerosis (ALS),…
Generation of clinical grade human iPS cells
The therapeutic use of stem cells depends on the availability of pluripotent cells that are not limited by technical, ethical or immunological considerations. The goal of this proposal is to…
Development of Induced Pluripotent Stem Cells for Modeling Human Disease
Human embryonic stem cells (hESC) hold great promise in regenerative medicine and cell replacement therapies because of their unique ability to self-renew and their developmental potential to form all cell…
hESC-Derived Motor Neurons For the Treatment of Cervical Spinal Cord Injury
Cervical spinal cord injuries result in a loss of upper limb function because the cells within the spinal cord that control upper limb muscles are destroyed. The goal of this…
Molecular and Cellular Transitions from ES Cells to Mature Functioning Human Neurons
Human embryonic stem cells (hESCs) are pluripotent entities, capable of generating a whole-body spectrum of distinct cell types. We have developmental procedures for inducing hESCs to develop into pure populations…
