The invention relates to si RNA molecules and their use in methods and pharmaceutical compositions for inhibiting the expression of the NRARP gene. The invention also relates to the use of said si RNAs molecules in the treatment and/or prevention of a disease or disorder related to neovascularization characterised by increased expression and/or activity of NRARP gene, said eye condition is selected from the group comprising age-related macular degeneration (AMD), ischemic retinopathy, diabetic macular edema (DME), proliferative diabetic retinopathy (PDR), diabetic retina ischemia (DRI), diabetic retinal edema (DRE) and retinopathy of prematurity (ROP) and combinations thereof.

The invention relates to iRNA, e.g., double stranded ribonucleic acid (dsRNA), compositions targeting the complement factor C3 gene, and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of a C3 gene and to treat subjects having a complement component C3-associated disease, e.g., paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), atypical hemolytic uremic syndrome (aHUS), neuromyelitis optica (NMO), multifocal motor neuropathy (MMN), myasthenia gravis (MG), and C3 glomerulonephritis.

The present invention provides novel bicyclic carbocyclic nucleosides and oligomeric compounds prepared therefrom. Incorporation of one or more of the bicyclic carbocyclic nucleosides into an oligomeric compound is expected to enhance one or more properties of the oligomeric compound. In certain embodiments, the oligomeric compounds provided herein hybridize to a portion of a target RNA resulting in modulation of normal function of the target RNA. In certain embodiments, bicyclic carbocyclic nucleosides are provided as monomers for use as antivirals.

CRISPR/Cas-related compositions and methods which provide for efficient gene editing of eukaryotic cells using modified gRNAs.

The present invention provides novel bicyclic carbocyclic nucleosides and oligomeric compounds prepared therefrom. Incorporation of one or more of the bicyclic carbocyclic nucleosides into an oligomeric compound is expected to enhance one or more properties of the oligomeric compound. In certain embodiments, the oligomeric compounds provided herein hybridize to a portion of a target RNA resulting in modulation of normal function of the target RNA. In certain embodiments, bicyclic carbocyclic nucleosides are provided as monomers for use as antivirals.

The present disclosure generally relates to systems, methods and compositions for use in Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-Cpf1 genome editing systems.

Disclosed are a carbocyclic nucleoside, an oligonucleotide and preparation methods therefor and medical uses thereof, and the carbocyclic nucleoside is a compound or salt shown in the following Formula (I). The oligonucleotide or pharmaceutically acceptable salt thereof provided in the present disclosure can be used for preparing a drug for gene therapy, gene vaccination, antisense therapy, short interfering RNA, or nucleic acid transfer. The oligonucleotide in the present disclosure has high binding affinity to single-stranded RNA, increase stability to nuclease degradation and plasma stability.

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