{"database":"GEO","file_versions":[{"headers":{"Content-Type":["application/json"]},"body":{"files":{"Other":["ftp://ftp.ncbi.nlm.nih.gov/geo/series/GSE320nnn/GSE320370/"]},"type":"primary"},"statusCode":"OK","statusCodeValue":200}],"scores":null,"additional":{"omics_type":["Other"],"species":["Homo sapiens"],"gds_type":["Other"],"full_dataset_link":["https://www.ncbi.nlm.nih.gov/geo/query/acc.cgi?acc=GSE320370"],"repository":["GEO"],"entry_type":["GSE"],"additional_accession":[]},"is_claimable":false,"name":"Tunable Gene Control via RNA Splicing with clinically approved small molecule","description":"Precise transgene regulation is crucial for safe and effective gene and cell therapies. Current inducible systems often rely on immunogenic exogenous proteins or non-clinically approved inducers, hindering clinical translation. We developed RisdiON, a compact inducible system controlled by risdiplam, a clinically approved oral drug that acts via splicing modulation. RisdiON utilizes risdiplam-responsive sequences for precise transgene control via endogenous splicing machinery, bypassing exogenous protein regulators, while its 'split-ATG' architecture ensures the expression of native, tag-free proteins. This approach provides robust, dose-dependent induction with minimal leakiness. RisdiON demonstrated broad utility, controlling various transgenes in immortalized cell lines and hiPSCs, enabling inducible CAR expression in primary T cells, and regulating Cas9 for precise gene editing. Additionally, adeno-associated virus (AAV)-delivered RisdiON achieved reversible transgene expression in vivo. The platform is modular and functional across diverse promoters, offering a safe, titratable, and reversible tool when coupled with an orally bioavailable drug to advance next-generation therapies.","dates":{"publication":"2026/04/30"},"accession":"GSE320370","cross_references":{"GSM":["GSM9541925","GSM9541926","GSM9541927","GSM9541928","GSM9541908","GSM9541922"],"GPL":["24676"],"GSE":["320370"],"taxon":["Homo sapiens"]}}