ArticleACS chemical biology2014
Genome editing-enabled HTS assays expand drug target pathways for Charcot-Marie-tooth disease.
Article in ACS chemical biology, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 24 papers.
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Who cites it
24 citing papers in PubMed, 34 citations in OpenAlex.
- Peripheral Myelin Protein-22 and Its Prominence in Charcot-Marie-Tooth Disease.Chemical reviews · 2026Review
- Tackling assay interference associated with small molecules.Nature reviews. Chemistry · 2024Review
- Novel Transmission-Blocking Antimalarials Identified by High-Throughput Screening of Plasmodium berghei Ookluc.Antimicrobial agents and chemotherapy · 2023Article
- qHTSWaterfall: 3-dimensional visualization software for quantitative high-throughput screening (qHTS) data.Journal of cheminformatics · 2023Article
- Trimethylene Methane Dianion Equivalent for the Asymmetric Consecutive Allylation of Aldehydes: Applications to Prins-Driven Macrocyclizations for the Synthesis of Bryostatin 1 and Analogues.The Journal of organic chemistry · 2022Article
- Genome-Edited Coincidence and PMP22-HiBiT Fusion Reporter Cell Lines Enable an Artifact-Suppressive Quantitative High-Throughput Screening Strategy forACS pharmacology & translational science · 2021Article
- Emerging Therapies for Charcot-Marie-Tooth Inherited Neuropathies.International journal of molecular sciences · 2021Review
- Function-Oriented Synthesis: Design, Synthesis, and Evaluation of Highly Simplified Bryostatin Analogues.The Journal of organic chemistry · 2020Article
- High-Throughput Screening to Identify Inhibitors of the Type I Interferon-Major Histocompatibility Complex Class I Pathway in Skeletal Muscle.ACS chemical biology · 2020Article
- Regulating PMP22 expression as a dosage sensitive neuropathy gene.Brain research · 2020Review
- Detecting Secretory Proteins by Acoustic Droplet Ejection in Multiplexed High-Throughput Applications.ACS chemical biology · 2019Article
- Doing it All - How Families are Reshaping Rare Disease Research.Pharmaceutical research · 2018Article
- Regulation of the neuropathy-associated Pmp22 gene by a distal super-enhancer.Human molecular genetics · 2018Article
- Myelin protein zero mutations and the unfolded protein response in Charcot Marie Tooth disease type 1B.Annals of clinical and translational neurology · 2018Article
- Genome-Edited Cell Lines for High-Throughput Screening.Methods in molecular biology (Clifton, N.J.) · 2018Article
- Molecular dynamics simulations reveal ligand-controlled positioning of a peripheral protein complex in membranes.Nature communications · 2017Article
- Tead1 regulates the expression of Peripheral Myelin Protein 22 during Schwann cell development.Human molecular genetics · 2016Article
- Advancing drug discovery for neuropsychiatric disorders using patient-specific stem cell models.Molecular and cellular neurosciences · 2016Review
- Thermodynamic Proxies to Compensate for Biases in Drug Discovery Methods.Pharmaceutical research · 2016Article
- Internal Ribosome Entry Site-Based Bicistronic In Situ Reporter Assays for Discovery of Transcription-Targeted Lead Compounds.Chemistry & biology · 2015Article
Corrections and comments
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Authors and funding
12 authors at 4 institutions in 1 country.
Funding
Abstract
Copy number variation resulting in excess PMP22 protein causes the peripheral neuropathy Charcot-Marie-Tooth disease, type 1A. To broadly interrogate chemically sensitive transcriptional pathways controlling PMP22 protein levels, we used the targeting precision of TALEN-mediated genome editing to embed reporters within the genetic locus harboring the Peripheral Myelin Protein 22 (Pmp22) gene. Using a Schwann cell line with constitutively high endogenous levels of Pmp22, we obtained allelic insertion of secreted bioluminescent reporters with sufficient signal to enable a 1536-well assay. Our findings from the quantitative high-throughput screening (qHTS) of several thousand drugs and clinically investigated compounds using this assay design both overlapped and expanded results from a previous assay using a randomly inserted reporter gene controlled by a single regulatory element of the Pmp22 gene. A key difference was the identification of a kinase-controlled inhibitory pathway of Pmp22 transcription revealed by the activity of the Protein kinase C (PKC)-modulator bryostatin.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.