"Compositions And Methods Comprising Engineered Short Nuclear Rna (Snrna)" in Patent Application Approval Process (USPTO 20230279397).

The RNA-targeting nucleic acid molecule of claim 2, wherein the target RNA of interest is a microsatellite repeat RNA. The method of claim 33, wherein the RNA-targeting nucleic acid molecule or AAV vector targets an RNA sequence encoding dystrophin (DMD). The RNA-targeting nucleic acid molecule of claim 1, wherein the esnRNA comprises a targeting sequence (TS) that targets a target RNA of interest. In some embodiments, the RNA-targeting nucleic acid molecule or AAV vector targets an RNA sequence encoding dystrophin (DMD). [Extracted from the article]

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