biostudies-literatureMirza MTelethonBeyond Batten Disease Foundatione859https://www.ebi.ac.uk/biostudies/studies/S-EPMC6900386biostudies-literatureUnknown7(12)<h4>Background</h4>One of the most important steps taken by Beyond Batten Disease Foundation in our quest to cure juvenile Batten (CLN3) disease is to understand the State of the Science. We believe that a strong understanding of where we are in our experimental understanding of the CLN3 gene, its regulation, gene product, protein structure, tissue distribution, biomarker use, and pathological responses to its deficiency, lays the groundwork for determining therapeutic action plans.<h4>Objectives</h4>To present an unbiased comprehensive reference tool of the experimental understanding of the CLN3 gene and gene product of the same name.<h4>Methods</h4>BBDF compiled all of the available CLN3 gene and protein data from biological databases, repositories of federally and privately funded projeMolecular genetics & genomic medicineThe CLN3 gene and protein: What we know.PMC6900386TGM16CBDM01Chandrachud USchmidtke CVainshtein AKerkovich DMHaslett LJDobzinski NMirza MPalmieri MNapolitano GDiRonza AGroh JStorch SfalseThe CLN3 gene and protein: What we know.<h4>Background</h4>One of the most important steps taken by Beyond Batten Disease Foundation in our quest to cure juvenile Batten (CLN3) disease is to understand the State of the Science. We believe that a strong understanding of where we are in our experimental understanding of the CLN3 gene, its regulation, gene product, protein structure, tissue distribution, biomarker use, and pathological responses to its deficiency, lays the groundwork for determining therapeutic action plans.<h4>Objectives</h4>To present an unbiased comprehensive reference tool of the experimental understanding of the CLN3 gene and gene product of the same name.<h4>Methods</h4>BBDF compiled all of the available CLN3 gene and protein data from biological databases, repositories of federally and privately funded proje2019-01-01T00:00:00Z2019 Dec2026-05-03T16:17:59.191Z2020-05-21T23:46:17ZS-EPMC69003863156871210.1002/mgg3.859