Human iPSC-based disease modeling studies identify a common mechanistic defect and potential therapies for AMD and related macular dystrophies.

Mendeley Data2026-04-09 收录

黄斑变性机制研究

iPSC疾病模型

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Raw uncropped western blot and gelatin zymography gel images from Main and Supplementary Figures

Genome wide analysis points towards subtype specific diseases in different genetic forms of Amyotrophic Lateral Sclerosis. Genome wide analysis points towards subtype specific diseases in different genetic forms of Amyotrophic Lateral Sclerosis

ALS疾病分子机制

iPSC疾病模型

We established iPSCs from healthy donors, FUS-ALS and SOD1-ALS patients. Using our differentiation protocol originally developed by Reinhardt et al.,2013, we diferentiated these iPSCs toward spinal mo

NIAID Data Ecosystem70

Additional file 1 of High-throughput screening identifies histone deacetylase inhibitors that modulate GTF2I expression in 7q11.23 microduplication autism spectrum disorder patient-derived cortical neurons

神经发育疾病药物筛选

iPSC疾病模型

Additional file 1: Table S1. Quality control of iPSC lines.

DataCite Commons2020-11-19 更新50

Discovery of Functionalized 1H‑Benzo[d]imidazoles That Confer Protective Effects in a Phenotypic CLN3 Disease Patient-Derived iPSC Model

神经退行性疾病药物

iPSC疾病模型

The neuronal ceroid lipofuscinoses (NCLs) are rare and fatal autosomal pediatric neurodegenerative disorders. The most prevalent subtype, CLN3, arises from a mutation in the CLN3 gene. Common phenotyp

Figshare30

File S1 - The Disease-Specific Phenotype in Cardiomyocytes Derived from Induced Pluripotent Stem Cells of Two Long QT Syndrome Type 3 Patients

iPSC疾病模型

心脏电生理学

The following files are included in File S1: Figure S1, Schematic of voltage-gated sodium channel structure and location of mutations described in this study. Figure S2, ECG analysis of the two LQTS-

NIAID Data Ecosystem60

Gene expression data from human fibroblasts and induced Pluripotent Stem cells from amyotrophic lateral sclerosis patients. Homo sapiens

ALS分子机制

iPSC疾病模型

We have generated human induced Pluripotent Stem cells (hiPSc) from amyotrophic lateral sclerosis (ALS, motor neuron disease) patients, using Sendai virus-mediated delivery of reprogramming factors. h

NIAID Data Ecosystem40

Human iPSC-based disease modeling studies identify a common mechanistic defect and potential therapies for AMD and related macular dystrophies.

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