OLIGONUCLEOTIDE COMPOSITIONS AND METHODS OF USE THEREOF

Among other things, the present disclosure provides RHO oligonucleotides, compositions, and methods. In some embodiments, provided oligonucleotides comprise nucleobase modifications, sugar modifications, internucleotidic linkage modifications and/or patterns thereof, and have improved properties, ac...

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Bibliographische Detailangaben
Hauptverfasser: IWAMOTO, Naoki, VATHIPADIEKAL, Vinod, BYRNE, Michael John, GUO, Lankai, VARGEESE, Chandra, HOSS, Andrew Guzior
Format: Patent
Sprache:eng ; fre ; ger
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Zusammenfassung:Among other things, the present disclosure provides RHO oligonucleotides, compositions, and methods. In some embodiments, provided oligonucleotides comprise nucleobase modifications, sugar modifications, internucleotidic linkage modifications and/or patterns thereof, and have improved properties, activities and/or selectivities. In some embodiments, the present disclosure provides RHO oligonucleotides, compositions and methods for preventing and/or treating RHO-related conditions, disorders or diseases, such as retinopathy (e.g, retinal degeneration, retinal degenerative disease, retinal degenerative disorder, inherited retinal degenerative disorder, retinitis pigmentosa, autosomal dominant retinitis pigmentosa, etc.).