Disease Focus: Neurological Disorders
Optimization of a human interneuron cell therapy for traumatic brain injury
Research Objective A cell therapy product comprised of inhibitory neurons that can migrate, integrate and restore neurologic function after traumatic brain injury. Impact Traumatic brain injury Major Proposed Activities Examine the most effective dose and safety profile of human iPSC-derived MGE cells grafted into rodent hippocampus. Determine whether human iPSC-derived MGE cells mature into appropriate […]
Development of a SYF2 antisense oligonucleotide (ASO) treatment for ALS
Research Objective We will develop an antisense oligonucleotide, or DNA therapy for diverse forms of amyotrophic lateral sclerosis (ALS). Impact ALS is fatal and incurable, and if successful, we will develop a treatment that slows or stops ALS progression across a broad range of patients. Major Proposed Activities Selection of the lead drug by testing […]
Transplantation of Pluripotent Stem Cell Derived Microglia for the Treatment of Adult-onset Leukoencephalopathy (HDLS/ALSP)
Research Objective We propose to investigate the transplantation of pluripotent stem cell derived microglia as a potential therapy for the devastating neurological disease; Adult-onset leukoencephalopathy (ALSP/HDLS). Impact The most immediately impacted condition will be ALSP. However, further examination of the safety of human microglial transplantation will have broad implications for many neurodegenerative disorders Major Proposed […]
Develop iPSC-derived microglia to treat progranulin-deficient Frontotemporal Dementia
Research Objective Develop stem cell-based therapy to treat dementia Impact There are no treatments for dementia. If successfully achieved, this study will lead to a cure of a familial
 form of dementia in the elderly population. Major Proposed Activities Develop a robust human stem cell-derived microglial platform for cell-based therapy Determine short-term safety and efficacy […]
Drug Development for Autism Spectrum Disorder Using Human Patient iPSCs
Research Objective We will use human patient induced pluripotent stem cell (hiPSC)-based models to screen for a drug that activates a transcription factor critical to the treatment of Autism Spectrum Disorder (ASD). Impact Our goal is to develop a small molecule to treat Autism Spectrum Disorder (ASD), which currently affects 1/68 children born in the […]
Generation and in vitro profiling of neural stem cell lines to predict in vivo efficacy for chronic cervical spinal cord injury.
Research Objective This project generates new cGMP compliant tissue educated human neural stem cell lines, paired with in vivo pre-clinical proof of concept testing, and development of a predictive in vitro profile. Impact Identification of new cell lines with in vivo efficacy testing to enable efficient translation to chronic cervical spinal cord injury, an area […]
iPS Glial Therapy for White Matter Stroke and Vascular Dementia
Research Objective This cell line will target recovery in ischemic white disease, a progressive dementing condition with no current therapy by developing a new stem line, iPS-glial enriched progenitors (iPS-GEPs). Impact This cell line will target tissue repair and recovery in ischemic white disease/vascular dementia, a chronically progressive and dementing condition with no current therapy. […]
Neural Stem Cell Relays for Severe Spinal Cord Injury
Research Objective We propose to utilize human neural stem cells to form neuronal relays across sites of severe SCI, restoring function across the site of spinal cord injury. Impact We will develop a specific type of neural stem cell that is best suited for repairing the injured spinal cord. Major Proposed Activities In Vitro Assessment […]
Development of a Cellular Therapeutic for Treatment of Epilepsy
Research Objective A stem cell-derived nerve cell therapy to minimize seizures in people with epilepsy Impact Many people with epilepsy have uncontrolled seizures that can be life threatening and adversely impact quality of life and independence. A cell therapy could help those not responsive to drugs. Major Proposed Activities Transplant a nerve cell therapy made […]
Identification and characterization of the optimal human neural stem cell line (hNSC) for the treatment of traumatic brain injury (TBI) 2.0.
Research Objective We propose to discover the optimal human neural stem cell candidate for traumatic brain injury. 4 hNSC products (2 ES derived & 2 fetal) will be compared with TBI/vehicle controls, & then each other. Impact Traumatic brain injury (TBI) affects more Americans than brain, breast, colon, lung and prostate cancer combined ! There […]