Gene therapy for retinal dystrophy
Counteracting splice defects in the CEP290 gene using RNA antisense oligonucleotides or Cas9-mediated gene editing is a therapeutic strategy for Leber congenital amaurosis type 10—a severe untreatable retinal dystrophy leading to childhood blindness.
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| Veröffentlicht in: | Nature Medicine 2019-02, Vol.25 (2), p.198-199 |
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| Hauptverfasser: | , |
| Format: | Artikel |
| Sprache: | eng |
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| Online-Zugang: | Volltext |
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| Zusammenfassung: | Counteracting splice defects in the
CEP290
gene using RNA antisense oligonucleotides or Cas9-mediated gene editing is a therapeutic strategy for Leber congenital amaurosis type 10—a severe untreatable retinal dystrophy leading to childhood blindness. |
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| ISSN: | 1078-8956 1546-170X 1744-7933 |
| DOI: | 10.1038/s41591-019-0346-1 |