{"doi":"10.1146/annurev-med-050715-104432","title":"Lysosomal Proteins as a Therapeutic Target in Neurodegeneration","abstract":"<jats:p> Several proteins that are mutated in lysosomal storage diseases are linked to neurodegenerative disease. This review focuses on some of these lysosomal enzymes and transporters, as well as current therapies that have emerged from the lysosomal storage disease field. Given the deeper genetic understanding of lysosomal defects in neurodegeneration, we explore why some of these orphan disease drug candidates are also attractive targets in subpopulations of individuals with neurodegenerative disease. </jats:p>","journal":"Annual Review of Medicine","year":2017,"id":14165,"datarank":0.9738061605355404,"base_score":3.4339872044851463,"endowment":3.4339872044851463,"self_citation_contribution":0.515098080672772,"citation_network_contribution":0.45870807986276835,"self_endowment_contribution":0.515098080672772,"citer_contribution":0.45870807986276835,"corpus_percentile":null,"corpus_rank":null,"citation_count":30,"citer_count":21,"citers_with_citation_signal":19,"citers_with_endowment":19,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":null,"is_oa":false,"file_count":0,"downloads":0,"has_version_chain":false,"published_date":null,"fair_score":null,"fair_percentile":null,"algorithm_id":"datarank_citation_only_1hop_v6","ranking_scope":"data_only","authors":[{"id":113129,"name":"Dimitri Krainc","orcid":null,"position":1,"is_corresponding":false},{"id":113128,"name":"Jessica M. Mc Donald","orcid":null,"position":0,"is_corresponding":false}],"reference_count":0,"raw_metadata":{"has_enrichment":true,"base_score":3.4339872044851463,"endowment":3.4339872044851463,"datacite_reuse_total":0,"file_count":0,"downloads":0,"views":0,"has_version_chain":false,"is_dataset":false,"is_oa":false,"pmid":"28099085","pmcid":null,"openalex_id":"https://openalex.org/W2466686196","authors":[],"funders":[],"total_grants":0,"fwci":2.361,"citation_percentile":0.88042253,"influential_citations":1,"citation_trend":[{"year":2018,"count":3},{"year":2019,"count":6},{"year":2020,"count":6},{"year":2022,"count":4},{"year":2023,"count":2},{"year":2024,"count":5},{"year":2025,"count":2},{"year":2026,"count":2}],"oa_status":"closed","license":null,"oa_locations":[{"url":"https://www.annualreviews.org/doi/pdf/10.1146/annurev-med-050715-104432","host_type":"publisher"},{"url":"https://doi.org/10.1146/annurev-med-050715-104432","host_type":"journal"},{"url":"https://pubmed.ncbi.nlm.nih.gov/28099085","host_type":"repository"}],"fields_of_study":["Lysosomal Storage Disorders Research","Cellular transport and secretion","Parkinson's Disease Mechanisms and Treatments","Medicine","Biology","Acetylglucosaminidase","Amyloidogenic Proteins","Autophagy","Carrier Proteins","Endocytosis","Glucosylceramidase","Hexosaminidase A","Hexosaminidase B","Humans","Intracellular Signaling Peptides and Proteins","Lysosomal Storage Diseases","Lysosomes","Membrane Glycoproteins","Neurodegenerative Diseases","Niemann-Pick C1 Protein","Proteins","Proton-Translocating ATPases","Sphingomyelin Phosphodiesterase"],"mesh_terms":["Niemann-Pick C1 Protein","Acetylglucosaminidase","Autophagy","Carrier Proteins","Endocytosis","Glucosylceramidase","Proton-Translocating ATPases","Humans","Lysosomes","Membrane Glycoproteins","Proteins","Sphingomyelin Phosphodiesterase","Lysosomal Storage Diseases","Neurodegenerative Diseases","Intracellular Signaling Peptides and Proteins","Hexosaminidase A","Hexosaminidase B","Amyloidogenic Proteins"],"keywords":["Neurodegeneration","Lysosomal storage disease","Disease","Lysosome","Biology","Lysosomal storage disorders","Drug discovery","Neuroscience","Bioinformatics","Medicine","Cell biology","Enzyme","Biochemistry","Pathology","Neurodegenerative disease","Parkinson's Disease","Neurodegenerative Drug Candidates"],"sdg_mappings":[{"sdg_number":0,"sdg_label":"Good health and well-being"}],"linked_datasets":[],"clinical_trials":[],"software_tools":[],"database_accessions":[],"source":"live","citation_network_status":"fetched"},"created_at":"2026-06-01T05:18:52.619936Z","pmid":null,"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":[]}