{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,20]],"date-time":"2026-05-20T05:54:01Z","timestamp":1779256441135,"version":"3.51.4"},"reference-count":59,"publisher":"MDPI AG","issue":"1","license":[{"start":{"date-parts":[[2022,1,5]],"date-time":"2022-01-05T00:00:00Z","timestamp":1641340800000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/100003914","name":"Kennedys Disease Association","doi-asserted-by":"publisher","award":["none"],"award-info":[{"award-number":["none"]}],"id":[{"id":"10.13039\/100003914","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100001691","name":"KAKENHI","doi-asserted-by":"publisher","award":["20H00527"],"award-info":[{"award-number":["20H00527"]}],"id":[{"id":"10.13039\/501100001691","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Genes"],"abstract":"<jats:p>The recent advances in nucleic acid therapeutics demonstrate the potential to treat hereditary neurological disorders by targeting their causative genes. Spinal and bulbar muscular atrophy (SBMA) is an X-linked and adult-onset neurodegenerative disorder caused by the expansion of trinucleotide cytosine-adenine-guanine repeats, which encodes a polyglutamine tract in the androgen receptor gene. SBMA belongs to the family of polyglutamine diseases, in which the use of nucleic acids for silencing a disease-causing gene, such as antisense oligonucleotides and small interfering RNAs, has been intensively studied in animal models and clinical trials. A unique feature of SBMA is that both motor neuron and skeletal muscle pathology contribute to disease manifestations, including progressive muscle weakness and atrophy. As both motor neurons and skeletal muscles can be therapeutic targets in SBMA, nucleic acid-based approaches for other motor neuron diseases and myopathies may further lead to the development of a treatment for SBMA. Here, we review studies of nucleic acid-based therapeutic approaches in SBMA and related neurological disorders and discuss current limitations and perspectives to apply these approaches to patients with SBMA.<\/jats:p>","DOI":"10.3390\/genes13010109","type":"journal-article","created":{"date-parts":[[2022,1,6]],"date-time":"2022-01-06T03:41:32Z","timestamp":1641440492000},"page":"109","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":13,"title":["Nucleic Acid-Based Therapeutic Approach for Spinal and Bulbar Muscular Atrophy and Related Neurological Disorders"],"prefix":"10.3390","volume":"13","author":[{"given":"Tomoki","family":"Hirunagi","sequence":"first","affiliation":[{"name":"Department of Neurology, Nagoya University Graduate School of Medicine, 65 Tsurumai-cho, Syowa-ku, Nagoya 466-8550, Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Kentaro","family":"Sahashi","sequence":"additional","affiliation":[{"name":"Department of Neurology, Nagoya University Graduate School of Medicine, 65 Tsurumai-cho, Syowa-ku, Nagoya 466-8550, Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Katherine G.","family":"Meilleur","sequence":"additional","affiliation":[{"name":"Research and Clinical Development, Neuromuscular Development Unit, Biogen, 300, Binney Street, Cambridge, MA 02142, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9453-9311","authenticated-orcid":false,"given":"Masahisa","family":"Katsuno","sequence":"additional","affiliation":[{"name":"Department of Neurology, Nagoya University Graduate School of Medicine, 65 Tsurumai-cho, Syowa-ku, Nagoya 466-8550, Japan"},{"name":"Department of Clinical Research Education, Nagoya University Graduate School of Medicine, 65 Tsurumai-cho, Syowa-ku, Nagoya 466-8550, Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"1968","published-online":{"date-parts":[[2022,1,5]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"671","DOI":"10.1212\/WNL.18.7.671","article-title":"Progressive proximal spinal and bulbar muscular atrophy of late onset. 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