Advancing ISP-001 for Mucopolysaccharidosis I: Clinical Expansion, Clinical Manufacturing and Release Testing

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• Post published:October 23, 2026
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Phase 1/2 Study to Evaluate the Safety, Tolerability, and Efficacy of MZ-1866, a gene therapy for Pitt Hopkins Syndrome

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• Post published:October 23, 2026
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Phase I/II Clinical Trial for CMT4J

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• Post published:October 23, 2026
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Personalized antisense oligonucleotide therapy for rare pediatric genetic disease: SCN2A

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• Post published:October 23, 2026
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A Phase I Open Label Study to Evaluate the Safety and Tolerability of ISP-001 in Patients with Mucopolysaccharidosis Type 1 and Advancing ISP-001 for Mucopolysaccharidosis I: Clinical Expansion, Clinical Manufacturing and Release Testing

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• Post published:October 23, 2026
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Systematic cell-type-resolved single-base resolution functional characterization of neurodevelopmental risk genes

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• Post published:October 23, 2026
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CRISPR/Cas9-mediated gene editing of Hematopoietic stem and progenitor cells for Friedreich’s ataxia

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• Post published:October 23, 2026
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Advancing Therapeutic Discovery Through Multimodal Analyses of Genetic and Cellular Mechanisms in Neuropsychiatric Disorders

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• Post published:October 23, 2026
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CIRM Center for Neuropsychiatric Stem Cell Proteomics

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• Post published:October 23, 2026
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Research Objective This project will interrogate interactions, distribution, and function of high-confidence neuropsychiatric disorder risk proteins, and identify convergent pathobiology of patient genetic variants. Impact Datasets and stem cell resources…

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Interrogation of tandem repeat variants contributing to neurodevelopmental and psychiatric traits using stem cell models

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• Post published:October 23, 2026
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Research Objective Our project will identify molecular and cellular changes induced by specific genetic variants implicated in schizophrenia and autism spectrum disorder in stem cells, neuroprogenitor cells and neurons. Impact…

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