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Cellular tools to study brain diseases affecting synaptic transmission

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  • Post published:May 20, 2025
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There is a group of brain diseases that are caused by functional abnormalities. The brains of patients afflicted with these diseases which include autism spectrum disorders, schizophrenia, depression, and mania…

Continue ReadingCellular tools to study brain diseases affecting synaptic transmission

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

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  • Post published:May 20, 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

Robust generation of induced pluripotent stem cells by a potent set of engineered factors

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  • Post published:May 20, 2025
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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…

Continue ReadingRobust generation of induced pluripotent stem cells by a potent set of engineered factors

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

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  • Post published:May 20, 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:May 20, 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:May 20, 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

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

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  • Post published:May 20, 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

Pluripotent and Somatic Stem Cell Models to Study Inherited Diarrheal Disorders

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  • Post published:May 20, 2025
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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.…

Continue ReadingPluripotent and Somatic Stem Cell Models to Study Inherited Diarrheal Disorders

Stem Cell Therapy for Duchenne Muscular Dystrophy

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  • Post published:May 20, 2025
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Duchenne muscular dystrophy (DMD) is the most common and serious form of muscular dystrophy. One out of every 3500 boys is born with the disorder, and it is invariably fatal.…

Continue ReadingStem Cell Therapy for Duchenne Muscular Dystrophy

Developing a therapeutic candidate for Canavan disease using induced pluripotent stem cell

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  • Post published:May 20, 2025
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Canavan disease is a devastating disease of infants which affects their neural development and leads to mental retardation and early death. It occurs in 1 in 6,400 persons in the…

Continue ReadingDeveloping a therapeutic candidate for Canavan disease using induced pluripotent stem cell
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