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Editing of Parkinson’s disease mutation in patient-derived iPSCs by zinc-finger nucleases

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  • Post published:December 13, 2025
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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…

Continue ReadingEditing of Parkinson’s disease mutation in patient-derived iPSCs by zinc-finger nucleases

Development and preclinical testing of new devices for cell transplantation to the brain.

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  • Post published:December 13, 2025
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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…

Continue ReadingDevelopment and preclinical testing of new devices for cell transplantation to the brain.

Developing a method for rapid identification of high-quality disease specific hIPSC lines

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  • Post published:December 13, 2025
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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…

Continue ReadingDeveloping a method for rapid identification of high-quality disease specific hIPSC lines

Use of hiPSCs to develop lead compounds for the treatment of genetic diseases

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  • Post published:December 13, 2025
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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…

Continue ReadingUse of hiPSCs to develop lead compounds for the treatment of genetic diseases

Development of small molecule screens for autism using patient-derived iPS cells

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  • Post published:December 13, 2025
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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…

Continue ReadingDevelopment of small molecule screens for autism using patient-derived iPS cells

Development of a Hydrogel Matrix for Stem Cell Growth and Neural Repair after Stroke

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  • Post published:December 13, 2025
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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…

Continue ReadingDevelopment of a Hydrogel Matrix for Stem Cell Growth and Neural Repair after Stroke

Site-specific integration of Lmx1a, FoxA2, & Otx2 to optimize dopaminergic differentiation

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  • Post published:December 13, 2025
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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…

Continue ReadingSite-specific integration of Lmx1a, FoxA2, & Otx2 to optimize dopaminergic differentiation

Inhibitory Nerve Cell Precursors: Dosing, Safety and Efficacy

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  • Post published:December 13, 2025
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Many neurological disorders are characterized by an imbalance between excitation and inhibition. Our ultimate goal: to develop a cell-based therapy to modulate aberrant brain activity in the treatment of these…

Continue ReadingInhibitory Nerve Cell Precursors: Dosing, Safety and Efficacy

Neural restricted, FAC-sorted, human neural stem cells to treat traumatic brain injury

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  • Post published:December 13, 2025
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Traumatic brain injury (TBI) affects 1.4 million Americans a year; 175,000 in California. When the brain is injured, nerve cells near the site of injury die due to the initial…

Continue ReadingNeural restricted, FAC-sorted, human neural stem cells to treat traumatic brain injury

Repair of Conus Medullaris/Cauda Equina Injury using Human ES Cell-Derived Motor Neurons

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  • Post published:December 13, 2025
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Injuries to the spinal cord commonly result from motor vehicle accidents, traumatic falls, diving, surfing, skiing, and snowboarding accidents, other forms of sports injuries, as well as from gunshot injuries…

Continue ReadingRepair of Conus Medullaris/Cauda Equina Injury using Human ES Cell-Derived Motor Neurons
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