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Bioinformatics Institute","Department of Biochemistry and Molecular Biology University of Southern Denmark"],"full_dataset_link":["https://www.ebi.ac.uk/ena/browser/view/PRJEB70285"],"broker_name":["ArrayExpress"],"long_description":["Cellular models of propionic acidemia were generated using HepG2 cells that were edited by CRISPR gene editing to introduce the pathogenic PCCA c.1285-1416A>G variant associated with propionic acidemia, and a 7 bp deletion of a predicted hnRNP A1 binding motif (c.1285-1411delTAGAACA), which causes complete and partial activation of an 84 bp pseudoexon from intron 14 in the PCCA gene, respectively. Treatment by transfection of splice-switching antisense oligonucleotides (SSO) to block inclusion of the PCCA pseudoexon in mRNA, was used to investigate this as a potential therapeutic strategy."],"repository":["ENA"],"additional_accession":[]},"is_claimable":false,"name":"RNA-seq of human cell line HepG2 edited with CRISPR and treated with splice-switching oligo inhibiting a pseudoexon in PCCA against control SSO and control HepG2 cells","description":"RNA-seq of human cell line HepG2 edited with CRISPR and treated with splice-switching oligo inhibiting a pseudoexon in PCCA against control SSO and control HepG2 cells","dates":{"last_updated":"2023-12-10","first_public":"2023-12-10"},"accession":"PRJEB70285","cross_references":{}}