ArticleAnnals of neurology2021
A Transcriptome-Based Drug Discovery Paradigm for Neurodevelopmental Disorders.
Article in Annals of neurology, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers.
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Who cites it
15 citing papers in PubMed, 21 citations in OpenAlex.
- Proteo-Metabolomic Profiling of PMM2-CDG Reveals Dysregulation of Retinoic Acid Synthesis, Myo-Inositol, and the Hexosamine Pathway.Journal of inherited metabolic disease · 2026Article
- Exploring Novel Indazole and Pyrazole Nucleoside Analogues: Enzymatic Synthesis, Characterization, and Biological Evaluation.International journal of molecular sciences · 2026Article
- Transcriptomic data and biomedical literature synergize in finding pharmacologic gene regulators.bioRxiv : the preprint server for biology · 2026Article
- Transcriptome-based screening in TARDBP/TDP-43 knock-in motor neurons identifies the NEDD8-activating enzyme inhibitor MLN4924.Scientific reports · 2025Article
- Diverse ancestral representation improves genetic intolerance metrics.Nature communications · 2025Article
- Transcriptomic analysis of rat prefrontal cortex following chronic stress induced by social isolation - Relevance to psychiatric and neurodevelopmental illness, and implications for treatment.Neurobiology of stress · 2024Article
- Curbing Rhes Actions: Mechanism-based Molecular Target for Huntington's Disease and Tauopathies.CNS & neurological disorders drug targets · 2024Review
- Neurodevelopmental deficits and cell-type-specific transcriptomic perturbations in a mouse model of HNRNPU haploinsufficiency.PLoS genetics · 2023Article
- Evidence of shared transcriptomic dysregulation of HNRNPU-related disorder between human organoids and embryonic mice.iScience · 2023Article
- Deciphering COVID-19 host transcriptomic complexity and variations for therapeutic discovery against new variants.iScience · 2022Article
- Cancer-driving mutations are enriched in genic regions intolerant to germline variation.Science advances · 2022Article
- Identification of the NRF2 transcriptional network as a therapeutic target for trigeminal neuropathic pain.Science advances · 2022Article
- Deficiency of autism-related Scn2a gene in mice disrupts sleep patterns and circadian rhythms.Neurobiology of disease · 2022Article
- How can same-gene mutations promote both cancer and developmental disorders?Science advances · 2022Article
- Making Sense of Patient-Derived iPSCs, Transdifferentiated Neurons, Olfactory Neuronal Cells, and Cerebral Organoids as Models for Psychiatric Disorders.The international journal of neuropsychopharmacology · 2021Review
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Authors and funding
5 authors at 2 institutions in 1 country.
Funding
Abstract
Advances in genetic discoveries have created substantial opportunities for precision medicine in neurodevelopmental disorders. Many of the genes implicated in these diseases encode proteins that regulate gene expression, such as chromatin-associated proteins, transcription factors, and RNA-binding proteins. The identification of targeted therapeutics for individuals carrying mutations in these genes remains a challenge, as the encoded proteins can theoretically regulate thousands of downstream targets in a considerable number of cell types. Here, we propose the application of a drug discovery approach originally developed for cancer called "transcriptome reversal" for these neurodevelopmental disorders. This approach attempts to identify compounds that reverse gene-expression signatures associated with disease states. ANN NEUROL 2021;89:199-211.
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