C - Chemistry – Metallurgy – 12 – N
Patent
C - Chemistry, Metallurgy
12
N
C12N 15/11 (2006.01) A61K 31/70 (2006.01) C12N 9/00 (2006.01) C12N 15/86 (2006.01) A61K 38/00 (2006.01)
Patent
CA 2287508
The subject invention provides materials and methods for efficient, specific reduction or elimination of unwanted mRNA. These materials and methods can be used in therapies for retinal diseases. In one embodiment, ribozymes which degrade mutant rod opsin mRNA are used to treat retinitis pigmentosa or diabetic retinopathy. In a further embodiment, ribozymes which cleave RNAs involved in nitric oxide activity are used to treat blood-retinal dysfonction. The preferred targets include VEGF, eNOS, and iNOS.
L'invention concerne des substances et des méthodes permettant une réduction ou une élimination spécifique et efficace d'ARNm indésirable. Ces substances et ces méthodes peuvent être utilisées dans le cadre de thérapies permettant de traiter des maladies de la rétine. Selon un mode de réalisation de l'invention, on utilise des ribozymes dégradant l'ARNm de rhodopsine mutant pour traiter la rétinite pigmentaire ou la rétinopathie diabétique. Selon un autre mode de réalisation, on utilise des ribozymes clivant les ARN impliqués dans l'activité de l'oxyde d'azote pour traiter des dysfonctions rétiniennes sanguines. Les cibles préférées sont notamment VEGF, eNOS et iNOS.
Drenser Kimberly
Hauswirth William W.
Lewin Alfred S.
Deeth Williams Wall Llp
The University Of Florida
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