{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,1]],"date-time":"2026-05-01T20:51:18Z","timestamp":1777668678147,"version":"3.51.4"},"reference-count":70,"publisher":"SAGE Publications","issue":"3-4","license":[{"start":{"date-parts":[[2025,2,1]],"date-time":"2025-02-01T00:00:00Z","timestamp":1738368000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Human Gene Therapy"],"published-print":{"date-parts":[[2025,2]]},"abstract":"<jats:p>\n                    Worldwide, thousands of male patients who carry ATP Binding Cassette Subfamily D Member 1 (\n                    <jats:italic toggle=\"yes\">ABCD1<\/jats:italic>\n                    ) mutations develop adrenomyeloneuropathy (AMN) in mid-adulthood, a debilitating axonopathy of the spinal cord. Today AAV gene therapy brings the most hope for this orphan disease. We previously reported that an AAV9-MAG-\n                    <jats:italic toggle=\"yes\">hABCD1<\/jats:italic>\n                    vector injected intravenously in the neonatal period prevented the disease in 2-year-old\n                    <jats:italic toggle=\"yes\">Abcd1\u2212\/\u2212<\/jats:italic>\n                    mice, the AMN mouse model. In the current study, the same vector was injected intracisternally at 18 months of age, when about half of\n                    <jats:italic toggle=\"yes\">Abcd1\u2212\/\u2212<\/jats:italic>\n                    mice start losing balance and motricity. As soon as 1\u20133 months after vector injection, motor tests have evolved differently in treated and untreated (UT) mice. Six months after vector, treated mice (\n                    <jats:italic toggle=\"yes\">n<\/jats:italic>\n                    = 24) had near-normal motor performances, whereas neurological state had deteriorated in UT mice (\n                    <jats:italic toggle=\"yes\">n<\/jats:italic>\n                    = 34). In five white matter regions of the cervical spinal cord,\n                    <jats:italic toggle=\"yes\">hABCD1<\/jats:italic>\n                    expression at 24 months of age was present in 22% (18\u201327) of oligodendrocytes (OLs) and 22% (17\u201326) of astrocytes and not detected in neurons or microglia. Abundant\n                    <jats:italic toggle=\"yes\">hABCD1<\/jats:italic>\n                    expression was also observed in OLs and astrocytes in the cerebellum and brainstem and, to a lesser level, in the lower spinal cord, not in the dorsal root ganglia or brain cortex. In conclusion, the effect of the AAV9-MAG-\n                    <jats:italic toggle=\"yes\">hABCD1<\/jats:italic>\n                    vector at an early symptomatic stage of the\n                    <jats:italic toggle=\"yes\">Abcd1\u2212\/\u2212<\/jats:italic>\n                    mouse model paves a new oligotropic way for the gene therapy of AMN.\n                  <\/jats:p>","DOI":"10.1089\/hum.2024.175","type":"journal-article","created":{"date-parts":[[2024,12,26]],"date-time":"2024-12-26T12:13:54Z","timestamp":1735215234000},"page":"88-100","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":2,"title":["Intracisternal AAV9-MAG-\n                    <i>hABCD1<\/i>\n                    Vector Reverses Motor Deficits in Adult Adrenomyeloneuropathy Mice"],"prefix":"10.1177","volume":"36","author":[{"given":"Yasemin","family":"\u00d6zg\u00fcr G\u00fcnes","sequence":"first","affiliation":[{"name":"Laboratoire des Maladies Neurod\u00e9g\u00e9n\u00e9ratives, MIRCen Institute, Fontenay-aux-Roses, France"},{"name":"NEURATRIS at MIRCen, Fontenay aux Roses, France"},{"name":"Horae Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, Massachusetts, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Catherine","family":"Le Stunff","sequence":"additional","affiliation":[{"name":"Laboratoire des Maladies Neurod\u00e9g\u00e9n\u00e9ratives, MIRCen Institute, Fontenay-aux-Roses, France"},{"name":"NEURATRIS at MIRCen, Fontenay aux Roses, France"},{"name":"UMR1195 Inserm and University Paris Saclay, Le Kremlin-Bic\u00eatre, France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0017-4572","authenticated-orcid":false,"given":"Pierre","family":"Bougn\u00e8res","sequence":"additional","affiliation":[{"name":"Laboratoire des Maladies Neurod\u00e9g\u00e9n\u00e9ratives, MIRCen Institute, Fontenay-aux-Roses, France"},{"name":"NEURATRIS at MIRCen, Fontenay aux Roses, France"},{"name":"Therapy Design Consulting, Vincennes, France"},{"name":"GETDOC, Groupe d\u2019\u00c9tudes Th\u00e9rapeutiques Diab\u00e8te-Ob\u00e9sit\u00e9-Croissance, Chaville, France."}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"179","published-online":{"date-parts":[[2025,2,10]]},"reference":[{"key":"e_1_3_5_2_2","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2021.293"},{"key":"e_1_3_5_3_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.ebiom.2023.104781"},{"key":"e_1_3_5_4_2","doi-asserted-by":"publisher","DOI":"10.1093\/jnen\/59.2.89"},{"key":"e_1_3_5_5_2","doi-asserted-by":"publisher","DOI":"10.1093\/brain\/aww068"},{"key":"e_1_3_5_6_2","doi-asserted-by":"publisher","DOI":"10.1212\/01.Wnl.0000164458.02141.06"},{"key":"e_1_3_5_7_2","volume-title":"Atlas of the Spinal Cord of the Rat, Mouse, Marmoset, Rhesus and Human","author":"Sengul G","year":"2012","unstructured":"Sengul G, , Watson C, , Tanaka I, et al. 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