{"doi":"10.1002/ana.25674","title":"Antisense Oligonucleotide Reverses Leukodystrophy in Canavan Disease Mice","abstract":"Marked elevation in the brain concentration of N-acetyl-L-aspartate (NAA) is a characteristic feature of Canavan disease, a vacuolar leukodystrophy resulting from deficiency of the oligodendroglial NAA-cleaving enzyme aspartoacylase. We now demonstrate that inhibiting NAA synthesis by intracisternal administration of a locked nucleic acid antisense oligonucleotide to young-adult aspartoacylase-deficient mice reverses their pre-existing ataxia and diminishes cerebellar and thalamic vacuolation and Purkinje cell dendritic atrophy. Ann Neurol 2020;87:480-485.","journal":"Annals of Neurology","year":2020,"id":96915,"datarank":0.0,"base_score":0.0,"endowment":0.0,"self_citation_contribution":0.0,"citation_network_contribution":0.0,"self_endowment_contribution":0.0,"citer_contribution":0.0,"corpus_percentile":null,"corpus_rank":null,"citation_count":33,"citer_count":0,"citers_with_citation_signal":0,"citers_with_endowment":0,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":0.9511,"is_data_producer":false,"deposit_databanks":null,"is_oa":true,"file_count":0,"downloads":0,"has_version_chain":false,"published_date":"2020-01-01","fair_score":null,"fair_percentile":null,"algorithm_id":"datarank_citation_only_1hop_v6","ranking_scope":"data_only","authors":[{"id":344282,"name":"Yan Wang","orcid":"0000-0002-7744-4078","position":1,"is_corresponding":false},{"id":479690,"name":"Travis Burns","orcid":"0000-0002-0795-1197","position":2,"is_corresponding":false},{"id":344279,"name":"Sheng Zhang","orcid":"0000-0002-0692-8999","position":3,"is_corresponding":false},{"id":479691,"name":"Sarah Sternbach","orcid":"0000-0002-5761-9512","position":4,"is_corresponding":false},{"id":479692,"name":"Jennifer McDonough","orcid":"0000-0001-9809-2289","position":5,"is_corresponding":false},{"id":344284,"name":"Fuzheng Guo","orcid":"0000-0003-3410-8389","position":6,"is_corresponding":false},{"id":479693,"name":"David Pleasure","orcid":"0000-0002-9122-1268","position":7,"is_corresponding":false},{"id":479689,"name":"Vanessa L. Hull","orcid":"0000-0003-3221-740X","position":0,"is_corresponding":true}],"reference_count":21,"raw_metadata":null,"created_at":"2026-07-18T22:35:06.014648Z","pmid":"31925837","pmcid":null,"fwci":null,"citation_percentile":null,"influential_citations":0,"oa_status":null,"license":null,"views":0,"total_file_size_bytes":0,"version_count":0,"fair_f":null,"fair_a":null,"fair_i":null,"fair_r":null,"fair_zscore":null,"fair_rationale":null,"fair_model":null,"fair_agent_version":null,"fair_fulltext_source":null,"fair_has_llm":null,"fair_computed_at":null,"clinical_trials":[],"software_tools":[],"db_accessions":[],"linked_datasets":[],"topics":[]}