Oligodendrocytes, the Forgotten Target of Gene Therapy
If the billions of oligodendrocytes (OLs) populating the central nervous system (CNS) of patients could express their feelings, they would undoubtedly tell gene therapists about their frustration with the other neural cell populations, neurons, microglia, or astrocytes, which have been the favorite...
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MDPI AG
2024-11-01
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| author | Yasemin Ozgür-Gunes Catherine Le Stunff Pierre Bougnères |
| author_facet | Yasemin Ozgür-Gunes Catherine Le Stunff Pierre Bougnères |
| author_sort | Yasemin Ozgür-Gunes |
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| description | If the billions of oligodendrocytes (OLs) populating the central nervous system (CNS) of patients could express their feelings, they would undoubtedly tell gene therapists about their frustration with the other neural cell populations, neurons, microglia, or astrocytes, which have been the favorite targets of gene transfer experiments. This review questions why OLs have been left out of most gene therapy attempts. The first explanation is that the pathogenic role of OLs is still discussed in most CNS diseases. Another reason is that the so-called ubiquitous CAG, CBA, CBh, or CMV promoters—widely used in gene therapy studies—are unable or poorly able to activate the transcription of episomal transgene copies brought by adeno-associated virus (AAV) vectors in OLs. Accordingly, transgene expression in OLs has either not been found or not been evaluated in most gene therapy studies in rodents or non-human primates. The aims of the current review are to give OLs their rightful place among the neural cells that future gene therapy could target and to encourage researchers to test the effect of OL transduction in various CNS diseases. |
| format | Article |
| id | doaj-art-9a4e8fb44ee444c282d0bce6d84b0a5d |
| institution | Kabale University |
| issn | 2073-4409 |
| language | English |
| publishDate | 2024-11-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Cells |
| spelling | doaj-art-9a4e8fb44ee444c282d0bce6d84b0a5d2024-12-13T16:24:11ZengMDPI AGCells2073-44092024-11-011323197310.3390/cells13231973Oligodendrocytes, the Forgotten Target of Gene TherapyYasemin Ozgür-Gunes0Catherine Le Stunff1Pierre Bougnères2Horae Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, MA 01605, USAMIRCen Institute, Laboratoire des Maladies Neurodégénératives, Commissariat à l’Energie Atomique, 92260 Fontenay-aux-Roses, FranceMIRCen Institute, Laboratoire des Maladies Neurodégénératives, Commissariat à l’Energie Atomique, 92260 Fontenay-aux-Roses, FranceIf the billions of oligodendrocytes (OLs) populating the central nervous system (CNS) of patients could express their feelings, they would undoubtedly tell gene therapists about their frustration with the other neural cell populations, neurons, microglia, or astrocytes, which have been the favorite targets of gene transfer experiments. This review questions why OLs have been left out of most gene therapy attempts. The first explanation is that the pathogenic role of OLs is still discussed in most CNS diseases. Another reason is that the so-called ubiquitous CAG, CBA, CBh, or CMV promoters—widely used in gene therapy studies—are unable or poorly able to activate the transcription of episomal transgene copies brought by adeno-associated virus (AAV) vectors in OLs. Accordingly, transgene expression in OLs has either not been found or not been evaluated in most gene therapy studies in rodents or non-human primates. The aims of the current review are to give OLs their rightful place among the neural cells that future gene therapy could target and to encourage researchers to test the effect of OL transduction in various CNS diseases.https://www.mdpi.com/2073-4409/13/23/1973AAVgene therapyoligodendrocytescentral nervous system |
| spellingShingle | Yasemin Ozgür-Gunes Catherine Le Stunff Pierre Bougnères Oligodendrocytes, the Forgotten Target of Gene Therapy Cells AAV gene therapy oligodendrocytes central nervous system |
| title | Oligodendrocytes, the Forgotten Target of Gene Therapy |
| title_full | Oligodendrocytes, the Forgotten Target of Gene Therapy |
| title_fullStr | Oligodendrocytes, the Forgotten Target of Gene Therapy |
| title_full_unstemmed | Oligodendrocytes, the Forgotten Target of Gene Therapy |
| title_short | Oligodendrocytes, the Forgotten Target of Gene Therapy |
| title_sort | oligodendrocytes the forgotten target of gene therapy |
| topic | AAV gene therapy oligodendrocytes central nervous system |
| url | https://www.mdpi.com/2073-4409/13/23/1973 |
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