{"id":"https://openalex.org/W2983175547","doi":"https://doi.org/10.1016/j.omtm.2019.10.011","title":"mRNA as a Novel Treatment Strategy for Hereditary Spastic Paraplegia Type 5","display_name":"mRNA as a Novel Treatment Strategy for Hereditary Spastic Paraplegia Type 5","publication_year":2019,"publication_date":"2019-11-03","ids":{"openalex":"https://openalex.org/W2983175547","doi":"https://doi.org/10.1016/j.omtm.2019.10.011","mag":"2983175547","pmid":"https://pubmed.ncbi.nlm.nih.gov/31828178","pmcid":"https://www.ncbi.nlm.nih.gov/pmc/articles/6888748"},"language":"en","primary_location":{"id":"doi:10.1016/j.omtm.2019.10.011","doi":"https://doi.org/10.1016/j.omtm.2019.10.011","is_oa":true,"landing_page_url":"https://doi.org/10.1016/j.omtm.2019.10.011","pdf_url":"http://www.cell.com/article/S2329050119301196/pdf","source":{"id":"https://openalex.org/S2764588101","display_name":"Molecular Therapy \u2014 Methods & Clinical Development","issn_l":"2329-0501","issn":["2329-0501"],"is_oa":true,"is_in_doaj":true,"is_core":true,"listed_in":["cwts-core","doaj","dongbi-d","ki-jl-1","poland-100","vabb-shw"],"host_organization":"https://openalex.org/P4310315673","host_organization_name":"Cell Press","host_organization_lineage":["https://openalex.org/P4310315673","https://openalex.org/P4310320990"],"host_organization_lineage_names":["Cell Press","Elsevier BV"],"type":"journal"},"license":"cc-by-nc-nd","license_id":"https://openalex.org/licenses/cc-by-nc-nd","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Molecular Therapy - Methods & Clinical Development","raw_type":"journal-article"},"volume":null,"type":"article","is_traditional":true,"indexed_in":["crossref","doaj","pubmed"],"open_access":{"is_oa":true,"oa_status":"gold","oa_url":"http://www.cell.com/article/S2329050119301196/pdf","any_repository_has_fulltext":true},"authorships":[{"author_position":"first","author":{"id":null,"display_name":"Stefan Hauser","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I2801533059","display_name":"German Center for Neurodegenerative Diseases","ror":"https://ror.org/043j0f473","country_code":"DE","type":"facility","lineage":["https://openalex.org/I1305996414","https://openalex.org/I2801533059","https://openalex.org/I4405256427"]},{"id":"https://openalex.org/I4210141865","display_name":"Hertie Institute for Clinical Brain Research","ror":"https://ror.org/04zzwzx41","country_code":"DE","type":"other","lineage":["https://openalex.org/I4210094118","https://openalex.org/I4210141865","https://openalex.org/I8087733"]},{"id":"https://openalex.org/I8087733","display_name":"University of T\u00fcbingen","ror":"https://ror.org/03a1kwz48","country_code":"DE","type":"education","lineage":["https://openalex.org/I8087733"]}],"countries":["DE"],"is_corresponding":true,"raw_author_name":"Stefan Hauser","raw_affiliation_strings":["Department of Neurology and Hertie Institute for Clinical Brain Research, University of T\u00fcbingen, 72076 T\u00fcbingen, Germany","German Center for Neurodegenerative Diseases (DZNE), 72076 T\u00fcbingen, Germany"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Neurology and Hertie Institute for Clinical Brain Research, University of T\u00fcbingen, 72076 T\u00fcbingen, Germany","institution_ids":["https://openalex.org/I4210141865","https://openalex.org/I8087733"]},{"raw_affiliation_string":"German Center for Neurodegenerative Diseases (DZNE), 72076 T\u00fcbingen, Germany","institution_ids":["https://openalex.org/I2801533059"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5036708417","display_name":"Marion Poenisch","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210123470","display_name":"CureVac (Germany)","ror":"https://ror.org/02q3n2241","country_code":"DE","type":"company","lineage":["https://openalex.org/I4210123470"]}],"countries":["DE"],"is_corresponding":false,"raw_author_name":"Marion Poenisch","raw_affiliation_strings":["CureVac AG, Paul-Ehrlich-Strasse 15, 72076 T\u00fcbingen, Germany"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"CureVac AG, Paul-Ehrlich-Strasse 15, 72076 T\u00fcbingen, Germany","institution_ids":["https://openalex.org/I4210123470"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5048887109","display_name":"Yvonne Schelling","orcid":"https://orcid.org/0009-0008-0787-0494","observed_orcids":["https://orcid.org/0009-0008-0787-0494"]},"institutions":[{"id":"https://openalex.org/I2801533059","display_name":"German Center for Neurodegenerative Diseases","ror":"https://ror.org/043j0f473","country_code":"DE","type":"facility","lineage":["https://openalex.org/I1305996414","https://openalex.org/I2801533059","https://openalex.org/I4405256427"]},{"id":"https://openalex.org/I4210141865","display_name":"Hertie Institute for Clinical Brain Research","ror":"https://ror.org/04zzwzx41","country_code":"DE","type":"other","lineage":["https://openalex.org/I4210094118","https://openalex.org/I4210141865","https://openalex.org/I8087733"]},{"id":"https://openalex.org/I8087733","display_name":"University of T\u00fcbingen","ror":"https://ror.org/03a1kwz48","country_code":"DE","type":"education","lineage":["https://openalex.org/I8087733"]}],"countries":["DE"],"is_corresponding":false,"raw_author_name":"Yvonne Schelling","raw_affiliation_strings":["Department of Neurology and Hertie Institute for Clinical Brain Research, University of T\u00fcbingen, 72076 T\u00fcbingen, Germany","German Center for Neurodegenerative Diseases (DZNE), 72076 T\u00fcbingen, Germany"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Neurology and Hertie Institute for Clinical Brain Research, University of T\u00fcbingen, 72076 T\u00fcbingen, Germany","institution_ids":["https://openalex.org/I4210141865","https://openalex.org/I8087733"]},{"raw_affiliation_string":"German Center for Neurodegenerative Diseases (DZNE), 72076 T\u00fcbingen, Germany","institution_ids":["https://openalex.org/I2801533059"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5078716419","display_name":"Philip H\u00f6flinger","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I2801533059","display_name":"German Center for Neurodegenerative Diseases","ror":"https://ror.org/043j0f473","country_code":"DE","type":"facility","lineage":["https://openalex.org/I1305996414","https://openalex.org/I2801533059","https://openalex.org/I4405256427"]},{"id":"https://openalex.org/I4210141865","display_name":"Hertie Institute for Clinical Brain Research","ror":"https://ror.org/04zzwzx41","country_code":"DE","type":"other","lineage":["https://openalex.org/I4210094118","https://openalex.org/I4210141865","https://openalex.org/I8087733"]},{"id":"https://openalex.org/I8087733","display_name":"University of T\u00fcbingen","ror":"https://ror.org/03a1kwz48","country_code":"DE","type":"education","lineage":["https://openalex.org/I8087733"]}],"countries":["DE"],"is_corresponding":false,"raw_author_name":"Philip H\u00f6flinger","raw_affiliation_strings":["Department of Neurology and Hertie Institute for Clinical Brain Research, University of T\u00fcbingen, 72076 T\u00fcbingen, Germany","German Center for Neurodegenerative Diseases (DZNE), 72076 T\u00fcbingen, Germany","Graduate School of Cellular and Molecular Neuroscience, University of T\u00fcbingen, 72076 T\u00fcbingen, Germany"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Neurology and Hertie Institute for Clinical Brain Research, University of T\u00fcbingen, 72076 T\u00fcbingen, Germany","institution_ids":["https://openalex.org/I4210141865","https://openalex.org/I8087733"]},{"raw_affiliation_string":"German Center for Neurodegenerative Diseases (DZNE), 72076 T\u00fcbingen, Germany","institution_ids":["https://openalex.org/I2801533059"]},{"raw_affiliation_string":"Graduate School of Cellular and Molecular Neuroscience, University of T\u00fcbingen, 72076 T\u00fcbingen, Germany","institution_ids":["https://openalex.org/I8087733"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5045998572","display_name":"Stefanie Schuster","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I2801533059","display_name":"German Center for Neurodegenerative Diseases","ror":"https://ror.org/043j0f473","country_code":"DE","type":"facility","lineage":["https://openalex.org/I1305996414","https://openalex.org/I2801533059","https://openalex.org/I4405256427"]},{"id":"https://openalex.org/I4210141865","display_name":"Hertie Institute for Clinical Brain Research","ror":"https://ror.org/04zzwzx41","country_code":"DE","type":"other","lineage":["https://openalex.org/I4210094118","https://openalex.org/I4210141865","https://openalex.org/I8087733"]},{"id":"https://openalex.org/I8087733","display_name":"University of T\u00fcbingen","ror":"https://ror.org/03a1kwz48","country_code":"DE","type":"education","lineage":["https://openalex.org/I8087733"]}],"countries":["DE"],"is_corresponding":false,"raw_author_name":"Stefanie Schuster","raw_affiliation_strings":["Department of Neurology and Hertie Institute for Clinical Brain Research, University of T\u00fcbingen, 72076 T\u00fcbingen, Germany","German Center for Neurodegenerative Diseases (DZNE), 72076 T\u00fcbingen, Germany","Graduate School of Cellular and Molecular Neuroscience, University of T\u00fcbingen, 72076 T\u00fcbingen, Germany"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Neurology and Hertie Institute for Clinical Brain Research, University of T\u00fcbingen, 72076 T\u00fcbingen, Germany","institution_ids":["https://openalex.org/I4210141865","https://openalex.org/I8087733"]},{"raw_affiliation_string":"German Center for Neurodegenerative Diseases (DZNE), 72076 T\u00fcbingen, Germany","institution_ids":["https://openalex.org/I2801533059"]},{"raw_affiliation_string":"Graduate School of Cellular and Molecular Neuroscience, University of T\u00fcbingen, 72076 T\u00fcbingen, Germany","institution_ids":["https://openalex.org/I8087733"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5036181854","display_name":"Axel Teegler","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210123470","display_name":"CureVac (Germany)","ror":"https://ror.org/02q3n2241","country_code":"DE","type":"company","lineage":["https://openalex.org/I4210123470"]}],"countries":["DE"],"is_corresponding":false,"raw_author_name":"Axel Teegler","raw_affiliation_strings":["CureVac AG, Paul-Ehrlich-Strasse 15, 72076 T\u00fcbingen, Germany"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"CureVac AG, Paul-Ehrlich-Strasse 15, 72076 T\u00fcbingen, Germany","institution_ids":["https://openalex.org/I4210123470"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5051583904","display_name":"Rabea Betten","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210123470","display_name":"CureVac (Germany)","ror":"https://ror.org/02q3n2241","country_code":"DE","type":"company","lineage":["https://openalex.org/I4210123470"]}],"countries":["DE"],"is_corresponding":false,"raw_author_name":"Rabea Betten","raw_affiliation_strings":["CureVac AG, Paul-Ehrlich-Strasse 15, 72076 T\u00fcbingen, Germany"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"CureVac AG, Paul-Ehrlich-Strasse 15, 72076 T\u00fcbingen, Germany","institution_ids":["https://openalex.org/I4210123470"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5110166525","display_name":"Jan-\u00c5ke Gustafsson","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I28166907","display_name":"Karolinska Institutet","ror":"https://ror.org/056d84691","country_code":"SE","type":"education","lineage":["https://openalex.org/I28166907"]},{"id":"https://openalex.org/I4387155253","display_name":"Center for Innovative Medicine","ror":"https://ror.org/00dgqhm63","country_code":"SE","type":"healthcare","lineage":["https://openalex.org/I1335113835","https://openalex.org/I28166907","https://openalex.org/I4387155253"]},{"id":"https://openalex.org/I44461941","display_name":"University of Houston","ror":"https://ror.org/048sx0r50","country_code":"US","type":"education","lineage":["https://openalex.org/I44461941"]}],"countries":["SE","US"],"is_corresponding":false,"raw_author_name":"Jan-\u00c5ke Gustafsson","raw_affiliation_strings":["Department of Biology and Biochemistry, Center for Nuclear Receptors and Cell Signaling, University of Houston, Houston, TX 77204, USA","Department of Biosciences and Nutrition, Center for Innovative Medicine, Karolinska Institutet, 141 83 Huddinge, Sweden"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Biology and Biochemistry, Center for Nuclear Receptors and Cell Signaling, University of Houston, Houston, TX 77204, USA","institution_ids":["https://openalex.org/I44461941"]},{"raw_affiliation_string":"Department of Biosciences and Nutrition, Center for Innovative Medicine, Karolinska Institutet, 141 83 Huddinge, Sweden","institution_ids":["https://openalex.org/I28166907","https://openalex.org/I4387155253"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5102780190","display_name":"Jeannette H\u00fcbener\u2010Schmid","orcid":"https://orcid.org/0000-0002-4973-0923","observed_orcids":["https://orcid.org/0000-0002-4973-0923"]},"institutions":[{"id":"https://openalex.org/I8087733","display_name":"University of T\u00fcbingen","ror":"https://ror.org/03a1kwz48","country_code":"DE","type":"education","lineage":["https://openalex.org/I8087733"]}],"countries":["DE"],"is_corresponding":false,"raw_author_name":"Jeannette H\u00fcbener-Schmid","raw_affiliation_strings":["Institute of Medical Genetics and Applied Genomics and Center of Rare Diseases, University of T\u00fcbingen, 72076 T\u00fcbingen, Germany"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Institute of Medical Genetics and Applied Genomics and Center of Rare Diseases, University of T\u00fcbingen, 72076 T\u00fcbingen, Germany","institution_ids":["https://openalex.org/I8087733"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5052221779","display_name":"Thomas Schlake","orcid":"https://orcid.org/0000-0001-6292-4055","observed_orcids":["https://orcid.org/0000-0001-6292-4055"]},"institutions":[{"id":"https://openalex.org/I4210123470","display_name":"CureVac (Germany)","ror":"https://ror.org/02q3n2241","country_code":"DE","type":"company","lineage":["https://openalex.org/I4210123470"]}],"countries":["DE"],"is_corresponding":false,"raw_author_name":"Thomas Schlake","raw_affiliation_strings":["CureVac AG, Paul-Ehrlich-Strasse 15, 72076 T\u00fcbingen, Germany"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"CureVac AG, Paul-Ehrlich-Strasse 15, 72076 T\u00fcbingen, Germany","institution_ids":["https://openalex.org/I4210123470"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5051948859","display_name":"Fr\u00e9d\u00e9ric Chevessier","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210123470","display_name":"CureVac (Germany)","ror":"https://ror.org/02q3n2241","country_code":"DE","type":"company","lineage":["https://openalex.org/I4210123470"]}],"countries":["DE"],"is_corresponding":false,"raw_author_name":"Fr\u00e9d\u00e9ric Chevessier-T\u00fcnnesen","raw_affiliation_strings":["CureVac AG, Paul-Ehrlich-Strasse 15, 72076 T\u00fcbingen, Germany"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"CureVac AG, Paul-Ehrlich-Strasse 15, 72076 T\u00fcbingen, Germany","institution_ids":["https://openalex.org/I4210123470"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5007498075","display_name":"Nigel Horscroft","orcid":"https://orcid.org/0000-0002-7313-2245","observed_orcids":["https://orcid.org/0000-0002-7313-2245"]},"institutions":[{"id":"https://openalex.org/I4210123470","display_name":"CureVac (Germany)","ror":"https://ror.org/02q3n2241","country_code":"DE","type":"company","lineage":["https://openalex.org/I4210123470"]}],"countries":["DE"],"is_corresponding":true,"raw_author_name":"Nigel Horscroft","raw_affiliation_strings":["CureVac AG, Paul-Ehrlich-Strasse 15, 72076 T\u00fcbingen, Germany"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"CureVac AG, Paul-Ehrlich-Strasse 15, 72076 T\u00fcbingen, Germany","institution_ids":["https://openalex.org/I4210123470"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5108028161","display_name":"Ingemar Bj\u00f6rkhem","orcid":"https://orcid.org/0000-0002-0575-9425","observed_orcids":["https://orcid.org/0000-0002-0575-9425"]},"institutions":[{"id":"https://openalex.org/I28166907","display_name":"Karolinska Institutet","ror":"https://ror.org/056d84691","country_code":"SE","type":"education","lineage":["https://openalex.org/I28166907"]}],"countries":["SE"],"is_corresponding":false,"raw_author_name":"Ingemar Bj\u00f6rkhem","raw_affiliation_strings":["Department of Laboratory Medicine, Karolinska Institutet, 171 77 Stockholm, Sweden"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Laboratory Medicine, Karolinska Institutet, 171 77 Stockholm, Sweden","institution_ids":["https://openalex.org/I28166907"]}]},{"author_position":"last","author":{"id":"https://openalex.org/A5076004371","display_name":"Ludger J. Sch\u00f6ls","orcid":"https://orcid.org/0000-0001-7774-5025","observed_orcids":["https://orcid.org/0000-0001-7774-5025"]},"institutions":[{"id":"https://openalex.org/I2801533059","display_name":"German Center for Neurodegenerative Diseases","ror":"https://ror.org/043j0f473","country_code":"DE","type":"facility","lineage":["https://openalex.org/I1305996414","https://openalex.org/I2801533059","https://openalex.org/I4405256427"]},{"id":"https://openalex.org/I4210141865","display_name":"Hertie Institute for Clinical Brain Research","ror":"https://ror.org/04zzwzx41","country_code":"DE","type":"other","lineage":["https://openalex.org/I4210094118","https://openalex.org/I4210141865","https://openalex.org/I8087733"]},{"id":"https://openalex.org/I8087733","display_name":"University of T\u00fcbingen","ror":"https://ror.org/03a1kwz48","country_code":"DE","type":"education","lineage":["https://openalex.org/I8087733"]}],"countries":["DE"],"is_corresponding":true,"raw_author_name":"Ludger Sch\u00f6ls","raw_affiliation_strings":["Department of Neurology and Hertie Institute for Clinical Brain Research, University of T\u00fcbingen, 72076 T\u00fcbingen, Germany","German Center for Neurodegenerative Diseases (DZNE), 72076 T\u00fcbingen, Germany"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Neurology and Hertie Institute for Clinical Brain Research, University of T\u00fcbingen, 72076 T\u00fcbingen, Germany","institution_ids":["https://openalex.org/I4210141865","https://openalex.org/I8087733"]},{"raw_affiliation_string":"German Center for Neurodegenerative Diseases (DZNE), 72076 T\u00fcbingen, Germany","institution_ids":["https://openalex.org/I2801533059"]}]}],"institutions":[],"countries_distinct_count":3,"institutions_distinct_count":7,"corresponding_author_ids":["https://openalex.org/A5007498075","https://openalex.org/A5076004371"],"corresponding_institution_ids":["https://openalex.org/I2801533059","https://openalex.org/I4210123470","https://openalex.org/I4210141865","https://openalex.org/I8087733"],"apc_list":{"value":3500,"currency":"USD","value_usd":3500},"apc_paid":{"value":3500,"currency":"USD","value_usd":3500},"fwci":1.4542,"has_fulltext":true,"cited_by_count":30,"citation_normalized_percentile":{"value":0.80928019,"is_in_top_1_percent":false,"is_in_top_10_percent":false},"cited_by_percentile_year":{"min":95,"max":99},"biblio":{"volume":"15","issue":null,"first_page":"359","last_page":"370"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T10725","display_name":"RNA Interference and Gene Delivery","score":0.6435999870300293,"subfield":{"id":"https://openalex.org/subfields/1312","display_name":"Molecular Biology"},"field":{"id":"https://openalex.org/fields/13","display_name":"Biochemistry, Genetics and Molecular Biology"},"domain":{"id":"https://openalex.org/domains/1","display_name":"Life Sciences"}},"topics":[{"id":"https://openalex.org/T10725","display_name":"RNA Interference and Gene Delivery","score":0.6435999870300293,"subfield":{"id":"https://openalex.org/subfields/1312","display_name":"Molecular Biology"},"field":{"id":"https://openalex.org/fields/13","display_name":"Biochemistry, Genetics and Molecular Biology"},"domain":{"id":"https://openalex.org/domains/1","display_name":"Life Sciences"}},{"id":"https://openalex.org/T12331","display_name":"Hereditary Neurological Disorders","score":0.1420000046491623,"subfield":{"id":"https://openalex.org/subfields/2804","display_name":"Cellular and Molecular Neuroscience"},"field":{"id":"https://openalex.org/fields/28","display_name":"Neuroscience"},"domain":{"id":"https://openalex.org/domains/1","display_name":"Life Sciences"}},{"id":"https://openalex.org/T10945","display_name":"Lysosomal Storage Disorders Research","score":0.05700000002980232,"subfield":{"id":"https://openalex.org/subfields/2716","display_name":"Genetics (clinical)"},"field":{"id":"https://openalex.org/fields/27","display_name":"Medicine"},"domain":{"id":"https://openalex.org/domains/4","display_name":"Health Sciences"}}],"keywords":[{"id":"https://openalex.org/keywords/cyp7b1","display_name":"CYP7B1","score":0.9279999732971191},{"id":"https://openalex.org/keywords/mrna-therapeutics","display_name":"mRNA therapeutics","score":0.8579999804496765},{"id":"https://openalex.org/keywords/oxysterols","display_name":"oxysterols","score":0.8100000023841858},{"id":"https://openalex.org/keywords/25-hydroxycholesterol","display_name":"25-hydroxycholesterol","score":0.9810000061988831},{"id":"https://openalex.org/keywords/27-hydroxycholesterol","display_name":"27-hydroxycholesterol","score":0.9990000128746033},{"id":"https://openalex.org/keywords/bile-acid-synthesis","display_name":"bile acid synthesis","score":0.8840000033378601},{"id":"https://openalex.org/keywords/neurodegeneration","display_name":"neurodegeneration","score":0.796999990940094},{"id":"https://openalex.org/keywords/blood-brain-barrier","display_name":"blood-brain barrier","score":0.7570000290870667},{"id":"https://openalex.org/keywords/pharmacokinetics","display_name":"pharmacokinetics","score":0.9290000200271606},{"id":"https://openalex.org/keywords/neurotoxicity","display_name":"neurotoxicity","score":0.6819999814033508},{"id":"https://openalex.org/keywords/gene-replacement","display_name":"gene replacement","score":0.45100000500679016}],"concepts":[{"id":"https://openalex.org/C2778111914","wikidata":"https://www.wikidata.org/wiki/Q7116159","display_name":"Oxysterol","level":3,"score":0.6774585843086243},{"id":"https://openalex.org/C2780395223","wikidata":"https://www.wikidata.org/wiki/Q657516","display_name":"Hereditary spastic paraplegia","level":4,"score":0.6565909385681152},{"id":"https://openalex.org/C105580179","wikidata":"https://www.wikidata.org/wiki/Q188928","display_name":"Messenger RNA","level":3,"score":0.49706318974494934},{"id":"https://openalex.org/C98274493","wikidata":"https://www.wikidata.org/wiki/Q128406","display_name":"Pharmacology","level":1,"score":0.4369838237762451},{"id":"https://openalex.org/C86803240","wikidata":"https://www.wikidata.org/wiki/Q420","display_name":"Biology","level":0,"score":0.4135923385620117},{"id":"https://openalex.org/C134018914","wikidata":"https://www.wikidata.org/wiki/Q162606","display_name":"Endocrinology","level":1,"score":0.3753131031990051},{"id":"https://openalex.org/C71924100","wikidata":"https://www.wikidata.org/wiki/Q11190","display_name":"Medicine","level":0,"score":0.36041608452796936},{"id":"https://openalex.org/C126322002","wikidata":"https://www.wikidata.org/wiki/Q11180","display_name":"Internal medicine","level":1,"score":0.338439404964447},{"id":"https://openalex.org/C104317684","wikidata":"https://www.wikidata.org/wiki/Q7187","display_name":"Gene","level":2,"score":0.27162742614746094},{"id":"https://openalex.org/C54355233","wikidata":"https://www.wikidata.org/wiki/Q7162","display_name":"Genetics","level":1,"score":0.13537877798080444},{"id":"https://openalex.org/C2778163477","wikidata":"https://www.wikidata.org/wiki/Q43656","display_name":"Cholesterol","level":2,"score":0.10563945770263672},{"id":"https://openalex.org/C127716648","wikidata":"https://www.wikidata.org/wiki/Q104053","display_name":"Phenotype","level":3,"score":0.10206568241119385}],"mesh":[],"locations_count":6,"locations":[{"id":"doi:10.1016/j.omtm.2019.10.011","doi":"https://doi.org/10.1016/j.omtm.2019.10.011","is_oa":true,"landing_page_url":"https://doi.org/10.1016/j.omtm.2019.10.011","pdf_url":"http://www.cell.com/article/S2329050119301196/pdf","source":{"id":"https://openalex.org/S2764588101","display_name":"Molecular Therapy \u2014 Methods & Clinical Development","issn_l":"2329-0501","issn":["2329-0501"],"is_oa":true,"is_in_doaj":true,"is_core":true,"listed_in":["cwts-core","doaj","dongbi-d","ki-jl-1","poland-100","vabb-shw"],"host_organization":"https://openalex.org/P4310315673","host_organization_name":"Cell Press","host_organization_lineage":["https://openalex.org/P4310315673","https://openalex.org/P4310320990"],"host_organization_lineage_names":["Cell Press","Elsevier BV"],"type":"journal"},"license":"cc-by-nc-nd","license_id":"https://openalex.org/licenses/cc-by-nc-nd","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Molecular Therapy - Methods & Clinical Development","raw_type":"journal-article"},{"id":"pmid:31828178","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/31828178","pdf_url":null,"source":{"id":"https://openalex.org/S4306525036","display_name":"PubMed","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I1299303238","host_organization_name":"National Institutes of Health","host_organization_lineage":["https://openalex.org/I1299303238"],"host_organization_lineage_names":[],"type":"repository"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Molecular therapy. Methods & clinical development","raw_type":"Journal Article"},{"id":"pmh:oai:pubmedcentral.nih.gov:6888748","doi":null,"is_oa":true,"landing_page_url":"https://pmc.ncbi.nlm.nih.gov/articles/PMC6888748/","pdf_url":null,"source":{"id":"https://openalex.org/S2764455111","display_name":"PubMed Central","issn_l":null,"issn":null,"is_oa":true,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I1299303238","host_organization_name":"National Institutes of Health","host_organization_lineage":["https://openalex.org/I1299303238"],"host_organization_lineage_names":[],"type":"repository"},"license":"cc-by-nc-nd","license_id":"https://openalex.org/licenses/cc-by-nc-nd","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Mol Ther Methods Clin Dev","raw_type":"Text"},{"id":"pmh:oai:doaj.org/article:4aeaa97d4bae48d099f191ccf0272017","doi":null,"is_oa":false,"landing_page_url":"https://doaj.org/article/4aeaa97d4bae48d099f191ccf0272017","pdf_url":null,"source":{"id":"https://openalex.org/S4306401280","display_name":"DOAJ (DOAJ: Directory of Open Access Journals)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":null,"host_organization_name":null,"host_organization_lineage":[],"host_organization_lineage_names":[],"type":"repository"},"license":null,"license_id":null,"version":"submittedVersion","is_accepted":false,"is_published":false,"raw_source_name":"Molecular Therapy: Methods & Clinical Development, Vol 15, Iss , Pp 359-370 (2019)","raw_type":"article"},{"id":"pmh:oai:opus.reutlingen-university.de:6217","doi":null,"is_oa":true,"landing_page_url":"https://publikationen.reutlingen-university.de/frontdoor/index/index/docId/6217","pdf_url":null,"source":{"id":"https://openalex.org/S4306400499","display_name":"Reutlingen University Academic Bibliography (Reutlingen University)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I57589565","host_organization_name":"Reutlingen University","host_organization_lineage":["https://openalex.org/I57589565"],"host_organization_lineage_names":[],"type":"repository"},"license":"cc-by-nc-nd","license_id":"https://openalex.org/licenses/cc-by-nc-nd","version":"submittedVersion","is_accepted":false,"is_published":false,"raw_source_name":null,"raw_type":"article"},{"id":"pmh:oai:pub.dzne.de:141657","doi":null,"is_oa":true,"landing_page_url":"https://pub.dzne.de/search?p=id:%22DZNE-2020-07981%22","pdf_url":null,"source":{"id":"https://openalex.org/S7407051172","display_name":"DZNE Pub","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I2801533059","host_organization_name":"German Center for Neurodegenerative Diseases","host_organization_lineage":["https://openalex.org/I2801533059"],"host_organization_lineage_names":[],"type":"repository"},"license":"other-oa","license_id":"https://openalex.org/licenses/other-oa","version":"submittedVersion","is_accepted":false,"is_published":false,"raw_source_name":"Molecular therapy 15, 359-370 (2019). doi:10.1016/j.omtm.2019.10.011","raw_type":"info:eu-repo/semantics/article"}],"best_oa_location":{"id":"doi:10.1016/j.omtm.2019.10.011","doi":"https://doi.org/10.1016/j.omtm.2019.10.011","is_oa":true,"landing_page_url":"https://doi.org/10.1016/j.omtm.2019.10.011","pdf_url":"http://www.cell.com/article/S2329050119301196/pdf","source":{"id":"https://openalex.org/S2764588101","display_name":"Molecular Therapy \u2014 Methods & Clinical Development","issn_l":"2329-0501","issn":["2329-0501"],"is_oa":true,"is_in_doaj":true,"is_core":true,"listed_in":["cwts-core","doaj","dongbi-d","ki-jl-1","poland-100","vabb-shw"],"host_organization":"https://openalex.org/P4310315673","host_organization_name":"Cell Press","host_organization_lineage":["https://openalex.org/P4310315673","https://openalex.org/P4310320990"],"host_organization_lineage_names":["Cell Press","Elsevier BV"],"type":"journal"},"license":"cc-by-nc-nd","license_id":"https://openalex.org/licenses/cc-by-nc-nd","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Molecular Therapy - Methods & Clinical Development","raw_type":"journal-article"},"sustainable_development_goals":[{"score":0.9940000176429749,"id":"https://metadata.un.org/sdg/3","display_name":"Good health and well-being"}],"study_designs":[],"awards":[],"funders":[{"id":"https://openalex.org/F4320306196","display_name":"Welch Foundation","ror":"https://ror.org/00np6vq88"}],"has_content":{"grobid_xml":true,"pdf":true},"content_urls":{"pdf":"https://content.openalex.org/works/W2983175547.pdf","grobid_xml":"https://content.openalex.org/works/W2983175547.grobid-xml"},"referenced_works_count":36,"referenced_works":["https://openalex.org/W64713844","https://openalex.org/W1420726270","https://openalex.org/W1538766821","https://openalex.org/W1963706689","https://openalex.org/W1973747307","https://openalex.org/W1988620094","https://openalex.org/W2014495927","https://openalex.org/W2039886688","https://openalex.org/W2040597914","https://openalex.org/W2068963865","https://openalex.org/W2082706052","https://openalex.org/W2088429050","https://openalex.org/W2092544712","https://openalex.org/W2094164512","https://openalex.org/W2104572662","https://openalex.org/W2115353736","https://openalex.org/W2123765156","https://openalex.org/W2133385892","https://openalex.org/W2147471451","https://openalex.org/W2160587950","https://openalex.org/W2162851037","https://openalex.org/W2171721352","https://openalex.org/W2266150566","https://openalex.org/W2280474488","https://openalex.org/W2474379397","https://openalex.org/W2587871737","https://openalex.org/W2742522266","https://openalex.org/W2767913965","https://openalex.org/W2772552632","https://openalex.org/W2777634859","https://openalex.org/W2782611922","https://openalex.org/W2807972887","https://openalex.org/W2899177904","https://openalex.org/W2920833479","https://openalex.org/W2922121037","https://openalex.org/W6752483878"],"related_works":["https://openalex.org/W4412458926","https://openalex.org/W2767913965","https://openalex.org/W4251979585","https://openalex.org/W2171721352","https://openalex.org/W1947649281","https://openalex.org/W2039886688","https://openalex.org/W2766225036","https://openalex.org/W2021129547","https://openalex.org/W2772552632","https://openalex.org/W2176400485"],"abstract_inverted_index":{"Hereditary":[0],"spastic":[1],"paraplegia":[2],"type":[3],"5":[4,136],"is":[5,63],"a":[6,104,120,152,181],"neurodegenerative":[7],"disease":[8],"caused":[9],"by":[10,68],"loss-of-function":[11],"mutations":[12],"in":[13,22,26,40,85,109,130,151,160,169],"the":[14,18,27,50,64,75,88,170,175],"CYP7B1":[15,31,67,83,100,125],"gene":[16],"encoding":[17],"oxysterol":[19,38],"7-\u03b1-hydroxylase":[20],"involved":[21],"bile":[23],"acid":[24],"synthesis":[25],"liver.":[28],"Lack":[29],"of":[30,36,66,70,78,95,107,116,123,140,155],"leads":[32],"to":[33,48,103,127,166,177,184],"an":[34],"accumulation":[35],"its":[37],"substrates,":[39],"particular":[41],"25-hydroxycholesterol":[42],"and":[43,53,81,111,149,162],"27-hydroxycholesterol":[44],"that":[45],"are":[46],"able":[47],"cross":[49],"blood-brain":[51],"barrier":[52],"have":[54],"neurotoxic":[55,156],"properties.":[56],"A":[57,92],"potential":[58,176],"therapeutic":[59],"strategy":[60],"for":[61,133,144],"SPG5":[62,188],"replacement":[65],"administration":[69],"mRNA.":[71],"Here,":[72],"we":[73],"studied":[74],"intravenous":[76],"application":[77],"formulated":[79],"mouse":[80,97],"human":[82,99,124],"mRNA":[84,101,126,141,179],"mice":[86],"lacking":[87],"endogenous":[89],"Cyp7b1":[90],"gene.":[91],"single-dose":[93],"injection":[94,122],"either":[96],"or":[98],"led":[102],"pronounced":[105],"degradation":[106],"oxysterols":[108,132,157],"liver":[110,161],"serum":[112,163],"within":[113],"2":[114],"days":[115,148],"treatment.":[117],"Pharmacokinetics":[118],"indicate":[119],"single":[121],"be":[128],"effective":[129],"reducing":[131],"at":[134,145],"least":[135,146],"days.":[137],"Repetitive":[138],"applications":[139],"were":[142],"safe":[143],"17":[147],"resulted":[150],"significant":[153],"reduction":[154],"not":[158],"only":[159],"but":[164],"also":[165],"some":[167],"extent":[168],"brain.":[171],"Our":[172],"study":[173],"highlights":[174],"use":[178],"as":[180],"novel":[182],"therapy":[183],"treat":[185],"patients":[186],"with":[187],"disease.":[189]},"counts_by_year":[{"year":2025,"cited_by_count":3},{"year":2024,"cited_by_count":4},{"year":2023,"cited_by_count":3},{"year":2022,"cited_by_count":8},{"year":2021,"cited_by_count":7},{"year":2020,"cited_by_count":5}],"updated_date":"2026-11-10T11:39:43.021295","created_date":"2019-11-22T00:00:00"}