{"doi":"10.1002/med.21705","title":"Modern approaches in gene therapy of motor neuron diseases","abstract":"<jats:title>Abstract</jats:title><jats:p>Motor neuron disorders are a group of neurodegenerative diseases characterized by muscle weakness, loss of ambulation, respiratory insufficiency, leading to an early death. Spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis are the most common and fatal motor neuron diseases. The last 3 years became very successful for novel gene therapy approaches in SMA in infants. Two innovative drugs—nusinersen (Spinraza) and onasemnogene abeparvovec (Zolgensma) have been approved by health authorities. The numerous molecular and genetic overlaps between different neurodegenerative diseases are of great importance in the development of innovative therapeutic strategies, including viral vector therapy and RNA modulating approaches.</jats:p>","journal":"Medicinal Research Reviews","year":2021,"id":19181,"datarank":0.8565450824703176,"base_score":3.1354942159291497,"endowment":3.1354942159291497,"self_citation_contribution":0.47032413238937254,"citation_network_contribution":0.38622095008094504,"self_endowment_contribution":0.47032413238937254,"citer_contribution":0.38622095008094504,"corpus_percentile":null,"corpus_rank":null,"citation_count":22,"citer_count":22,"citers_with_citation_signal":14,"citers_with_endowment":14,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":null,"is_data_producer":false,"deposit_databanks":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":130727,"name":"Maria Zakharova","orcid":"0000-0002-1072-9968","position":0,"is_corresponding":false}],"reference_count":0,"raw_metadata":{"has_enrichment":true,"base_score":3.1354942159291497,"endowment":3.1354942159291497,"datacite_reuse_total":0,"file_count":0,"downloads":0,"views":0,"has_version_chain":false,"is_dataset":false,"is_oa":false,"pmid":"32638429","pmcid":null,"openalex_id":"https://openalex.org/W3041476542","authors":[],"funders":[],"total_grants":0,"fwci":1.2603,"citation_percentile":0.79853723,"influential_citations":0,"citation_trend":[{"year":2021,"count":4},{"year":2022,"count":2},{"year":2023,"count":4},{"year":2024,"count":5},{"year":2025,"count":3},{"year":2026,"count":4}],"oa_status":"closed","license":"http://onlinelibrary.wiley.com/termsAndConditions#vor","oa_locations":[{"url":"https://api.wiley.com/onlinelibrary/tdm/v1/articles/10.1002%2Fmed.21705","host_type":"publisher"},{"url":"https://onlinelibrary.wiley.com/doi/pdf/10.1002/med.21705","host_type":"publisher"},{"url":"https://onlinelibrary.wiley.com/doi/full-xml/10.1002/med.21705","host_type":"publisher"},{"url":"https://doi.org/10.1002/med.21705","host_type":"journal"},{"url":"https://pubmed.ncbi.nlm.nih.gov/32638429","host_type":"repository"}],"fields_of_study":["Neurogenetic and Muscular Disorders Research","Genetics and Neurodevelopmental Disorders","Muscle Physiology and Disorders","Medicine","Amyotrophic Lateral Sclerosis","Genetic Therapy","Genetic Vectors","Humans","Infant","Muscular Atrophy, Spinal","Neurodegenerative Diseases"],"mesh_terms":["Amyotrophic Lateral Sclerosis","Genetic Vectors","Humans","Infant","Muscular Atrophy, Spinal","Genetic Therapy","Neurodegenerative Diseases"],"keywords":["Spinal muscular atrophy","Amyotrophic lateral sclerosis","SMA*","Motor neuron","Medicine","Genetic enhancement","Neuroscience","Weakness","Atrophy","Bioinformatics","Progressive muscular atrophy","Physical medicine and rehabilitation","Disease","Gene","Pathology","Biology","Genetics","Surgery","Gene therapy","Viral Vectors","Motor Neuron Diseases","Antisense Oligonucleotide Therapy"],"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":"2026-06-04T02:26:55.166269Z","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":[]}