{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"omics_type":["Unknown"],"volume":["12(1)"],"submitter":["Graceffa V"],"funding":["Cystinosis Ireland"],"pubmed_abstract":["Cystinosis is a rare disease, caused by a mutation in the gene cystinosin and characterised by the accumulation of cystine crystals. Advantages of biomaterial-mediated gene delivery include reduced safety concerns and the possibility to cure organs that are difficult to treat using systemic gene transfer methods. This study developed novel fibrin hydrogels for controlled, localised gene delivery, for the treatment of cystinosis. In the first part, fabrication parameters (i.e., DNA, thrombin, and aprotinin concentrations) were optimised, using a Design of Experiment (DOE) methodology. DOE is a statistical engineering approach to process optimisation, which increases experimental efficiency, reduces the number of experiments, takes into consideration interactions between different parameters"],"journal":["Scientific reports"],"pagination":["3752"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC8904479"],"repository":["biostudies-literature"],"pubmed_title":["Development of a fibrin-mediated gene delivery system for the treatment of cystinosis via design of experiment."],"pmcid":["PMC8904479"],"pubmed_authors":["Graceffa V"],"additional_accession":[]},"is_claimable":false,"name":"Development of a fibrin-mediated gene delivery system for the treatment of cystinosis via design of experiment.","description":"Cystinosis is a rare disease, caused by a mutation in the gene cystinosin and characterised by the accumulation of cystine crystals. Advantages of biomaterial-mediated gene delivery include reduced safety concerns and the possibility to cure organs that are difficult to treat using systemic gene transfer methods. This study developed novel fibrin hydrogels for controlled, localised gene delivery, for the treatment of cystinosis. In the first part, fabrication parameters (i.e., DNA, thrombin, and aprotinin concentrations) were optimised, using a Design of Experiment (DOE) methodology. DOE is a statistical engineering approach to process optimisation, which increases experimental efficiency, reduces the number of experiments, takes into consideration interactions between different parameters","dates":{"release":"2022-01-01T00:00:00Z","publication":"2022 Mar","modification":"2025-04-05T11:07:31.954Z","creation":"2025-04-05T11:07:31.954Z"},"accession":"S-EPMC8904479","cross_references":{"pubmed":["35260693"],"doi":["10.1038/s41598-022-07750-y"]}}