{"doi":"10.1016/j.pharmr.2025.100093","title":"Genetic therapies for neurological diseases","abstract":"Often, gene therapy reviews concentrate upon specific therapeutic modalities-particularly either viral vector-mediated or a nonviral approach. Here, we draw together a comprehensive array of knowledge across the field of genetic therapy for genetic neurological disease. The sections on preclinical and clinical application of viral vectors are followed by sections on RNA-based therapies and then by antisense oligonucleotide approaches also in preclinical and clinical settings. We present a separate section on gene editing strategies and conclude with a section elaborating on the neurosurgical techniques and the expertise required for clinical application of many of these technologies. SIGNIFICANCE STATEMENT: Genetic therapies have significant potential to treat life-limiting neurological diseases. This review examines the different approaches, clinical successes, and considerations on how to deploy them.","journal":"Pharmacological Reviews","year":2025,"id":534923,"datarank":0.20794415416798362,"base_score":1.3862943611198906,"endowment":1.3862943611198906,"self_citation_contribution":0.20794415416798362,"citation_network_contribution":0.0,"self_endowment_contribution":0.20794415416798362,"citer_contribution":0.0,"corpus_percentile":null,"corpus_rank":null,"citation_count":3,"citer_count":3,"citers_with_citation_signal":0,"citers_with_endowment":0,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":0.9471,"is_data_producer":true,"deposit_databanks":{"ClinicalTrials.gov":["NCT04539041"]},"is_oa":true,"file_count":0,"downloads":0,"has_version_chain":false,"published_date":"2025-01-01","fair_score":null,"fair_percentile":null,"algorithm_id":"datarank_citation_only_1hop_v6","ranking_scope":"data_only","authors":[{"id":397614,"name":"Manju A. Kurian","orcid":"0000-0003-3529-5075","position":1,"is_corresponding":false},{"id":439326,"name":"Haiyan Zhou","orcid":"0000-0001-8440-3074","position":2,"is_corresponding":false},{"id":1418061,"name":"Ross Ferguson","orcid":"0009-0000-9588-2656","position":3,"is_corresponding":false},{"id":809073,"name":"Sarah J. Tabrizi","orcid":"0000-0003-2716-2045","position":4,"is_corresponding":false},{"id":1161479,"name":"Gabriele Lignani","orcid":"0000-0002-3963-9296","position":5,"is_corresponding":false},{"id":236639,"name":"Kristian Aquilina","orcid":"0000-0001-7015-5196","position":6,"is_corresponding":false},{"id":461427,"name":"Simon N. Waddington","orcid":"0000-0003-4970-4730","position":7,"is_corresponding":false},{"id":1056513,"name":"Ahad A. Rahim","orcid":"0000-0003-0044-0949","position":0,"is_corresponding":true}],"reference_count":358,"raw_metadata":null,"created_at":"2026-07-19T02:51:52.019261Z","pmid":"41389437","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":[]}