Long-Term Disease Prevention with a Gene Therapy Targeting Oligodendrocytes in a Mouse Model of Adrenomyeloneuropathy.

Özgür-Günes, Yasemin; Chedik, Malha; Le Stunff, Catherine; Fovet, Claire-Maëlle; Bougnères, Pierre

Özgür-Günes, Yasemin; Chedik, Malha; Le Stunff, Catherine; Fovet, Claire-Maëlle; Bougnères, Pierre.

Affiliation

Özgür-Günes Y; UMR1195 Inserm and University Paris Saclay, Le Kremlin-Bicêtre, France.
Chedik M; UMR1195 Inserm and University Paris Saclay, Le Kremlin-Bicêtre, France.
Le Stunff C; UMR1195 Inserm and University Paris Saclay, Le Kremlin-Bicêtre, France.
Fovet CM; MIRCen Institute, CEA, Fontenay-aux-Roses, France.
Bougnères P; UMR1195 Inserm and University Paris Saclay, Le Kremlin-Bicêtre, France.

Hum Gene Ther ; 33(17-18): 936-949, 2022 Sep.

Article in En | MEDLINE | ID: mdl-35166123

Subject(s)

Adrenoleukodystrophy; ATP Binding Cassette Transporter, Subfamily D, Member 1/genetics; Adrenoleukodystrophy/genetics; Adrenoleukodystrophy/therapy; Animals; Disease Models, Animal; Fatty Acids/metabolism; Genetic Therapy; Humans; Mice; Myelin-Associated Glycoprotein/genetics; Myelin-Associated Glycoprotein/metabolism; Oligodendroglia/metabolism

Key words

AAV9; adrenomyeloneuropathy; gene therapy; oligodendrocytes

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Full text: 1 Database: MEDLINE Main subject: Adrenoleukodystrophy Limits: Animals / Humans Language: En Year: 2022 Type: Article

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Full text: 1 Database: MEDLINE Main subject: Adrenoleukodystrophy Limits: Animals / Humans Language: En Year: 2022 Type: Article