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Long-Term Disease Prevention with a Gene Therapy Targeting Oligodendrocytes in a Mouse Model of Adrenomyeloneuropathy.
Hum Gene Ther. 2022 Sep;33(17-18):936-949. doi: 10.1089/hum.2021.293. Epub 2022 Jul 18.
Hum Gene Ther. 2022.
PMID: 35166123
Correction of a knock-in mouse model of acrodysostosis with gene therapy using a rAAV9-CAG-human PRKAR1A vector.
Özgür-Günes Y, Le Stunff C, Chedik M, Belot MP, Becker PH, Blouin V, Bougnères P.
Özgür-Günes Y, et al.
Gene Ther. 2022 Aug;29(7-8):441-448. doi: 10.1038/s41434-021-00286-2. Epub 2021 Oct 1.
Gene Ther. 2022.
PMID: 34599290
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