Derivation: (2024) ATM-deficiency-induced microglial activation promotes neurodegeneration in ataxia-telangiectasia (2023) Imbalanced unfolded protein response signaling contributes to 1-deoxysphingolipid retinal toxicity (2023) Single‐cell transcriptomics stratifies organoid models of metabolic dysfunction‐associated steatotic liver disease (2023) Polystyrene nanoplastics affect transcriptomic and epigenomic signatures of human fibroblasts and derived induced pluripotent stem cells: Implications for human health (2023) iPSC–derived retinal pigmented epithelial cells from patients with macular telangiectasia show decreased mitochondrial function (2023) Integrating human iPSC‐derived macrophage progenitors into retinal organoids to generate a mature retinal microglial niche (2023) Increased degradation of FMRP contributes to neuronal hyperexcitability in tuberous sclerosis complex (2022) Molecular convergence between Down syndrome and fragile X syndrome identified using human pluripotent stem cell models (2022) The 22q11. 2 region regulates presynaptic gene-products linked to schizophrenia (2022) Cell-specific cis-regulatory elements and mechanisms of non-coding genetic disease in human retina and retinal organoids (2022) Patient-derived three-dimensional cortical neurospheres to model Parkinson's disease (2022) Attenuation of SARS-CoV-2 infection by losartan in human kidney organoids (2022) 16p13. 11 deletion variants associated with neuropsychiatric disorders cause morphological and synaptic changes in induced pluripotent stem cell-derived neurons (2022) A Brain-Penetrant Stearoyl-CoA Desaturase Inhibitor Reverses α-Synuclein Toxicity (2022) Allele-specific silencing of the gain-of-function mutation in Huntington's disease using CRISPR/Cas9 (2022) Extra-hematopoietic immunomodulatory role of the guanine-exchange factor DOCK2 (2022) Angelman syndrome patient neuron screen identifies a potent and selective clinical ASO targeting UBE3A-ATS with long lasting effect in cynomolgus monkey Genome Editing: (2023) Directed differentiation of human pluripotent stem cells into articular cartilage reveals effects caused by absence of WISP3, the gene responsible for progressive pseudorheumatoid arthropathy of childhood (2022) DGAT2 inhibition potentiates lipid droplet formation to reduce cytotoxicity in APOL1 kidney risk variants (2023) Cell-type specific defects in PTEN-mutant cortical organoids converge on abnormal circuit activity (2023) Human pluripotent stem cells-derived inner ear organoids recapitulate otic development in vitro. (2022) Autism genes converge on asynchronous development of shared neuron classes
