{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,30]],"date-time":"2026-09-30T16:21:29Z","timestamp":1790785289035,"version":"4.1.0"},"reference-count":147,"publisher":"MDPI AG","issue":"18","license":[{"start":{"date-parts":[[2019,9,6]],"date-time":"2019-09-06T00:00:00Z","timestamp":1567728000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["IJMS"],"abstract":"<jats:p>Amyotrophic lateral sclerosis (ALS) is a fatal motor neuron disease (MND) with no cure. Recent advances in gene therapy open a new perspective to treat this disorder\u2014particularly for the characterized genetic forms. Gene therapy approaches, involving the delivery of antisense oligonucleotides into the central nervous system (CNS) are being tested in clinical trials for patients with mutations in SOD1 or C9orf72 genes. Viral vectors can be used to deliver therapeutic sequences to stably transduce motor neurons in the CNS. Vectors derived from adeno-associated virus (AAV), can efficiently target genes and have been tested in several pre-clinical settings with promising outcomes. Recently, the Food and Drug Administration (FDA) approved Zolgensma, an AAV-mediated treatment for another MND\u2014the infant form of spinal muscular atrophy. Given the accelerated progress in gene therapy, it is potentially a promising avenue to develop an efficient and safe cure for ALS.<\/jats:p>","DOI":"10.3390\/ijms20184388","type":"journal-article","created":{"date-parts":[[2019,9,9]],"date-time":"2019-09-09T03:14:41Z","timestamp":1567998881000},"page":"4388","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":92,"title":["Gene Therapy for ALS\u2014A Perspective"],"prefix":"10.3390","volume":"20","author":[{"given":"Marisa","family":"Cappella","sequence":"first","affiliation":[{"name":"Sorbonne Universit\u00e9, Inserm UMRS 974, Centre of Research in Myology (CRM), Institut de Myologie, GH Piti\u00e9 Salp\u00eatri\u00e8re, 75013 Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chiara","family":"Ciotti","sequence":"additional","affiliation":[{"name":"Sorbonne Universit\u00e9, Inserm UMRS 974, Centre of Research in Myology (CRM), Institut de Myologie, GH Piti\u00e9 Salp\u00eatri\u00e8re, 75013 Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mathilde","family":"Cohen-Tannoudji","sequence":"additional","affiliation":[{"name":"Sorbonne Universit\u00e9, Inserm UMRS 974, Centre of Research in Myology (CRM), Institut de Myologie, GH Piti\u00e9 Salp\u00eatri\u00e8re, 75013 Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maria Grazia","family":"Biferi","sequence":"additional","affiliation":[{"name":"Sorbonne Universit\u00e9, Inserm UMRS 974, Centre of Research in Myology (CRM), Institut de Myologie, GH Piti\u00e9 Salp\u00eatri\u00e8re, 75013 Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2019,9,6]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"942","DOI":"10.1016\/S0140-6736(10)61156-7","article-title":"Amyotrophic lateral sclerosis","volume":"377","author":"Kiernan","year":"2011","journal-title":"Lancet"},{"key":"ref_2","doi-asserted-by":"crossref","first-page":"735","DOI":"10.1016\/j.ncl.2015.07.006","article-title":"Patterns of Weakness, Classification of Motor Neuron Disease, and Clinical Diagnosis of Sporadic Amyotrophic Lateral Sclerosis","volume":"33","author":"Statland","year":"2015","journal-title":"Neurol. 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