C12N2310/319

TREATMENT OF COLLAGEN GENE RELATED DISEASES BY INHIBITION OF NATURAL ANTISENSE TRANSCRIPT TO A COLLAGEN GENE
20170073680 · 2017-03-16 ·

The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of a Collagen gene, in particular, by targeting natural antisense polynucleotides of a Collagen gene. The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of Collagen genes.

Synthetic lariat RNA for RNA interference

Provided herein are compositions for the modification of gene expression by RNA interference using a microRNA (miRNA) guide strand precursor. Methods of making and using the miRNA guide strand precursor also are provided. The compositions bypass the Drosha-Exportin-Dicer pathway and are resistant to nucleases.

REVERSIR TM COMPOUNDS

The present invention relates, in general to agents that modulate the pharmacological activity of conjugated siRNAs.

SYNTHESIS OF 3 -RNA OLIGONUCLEOTIDES

The disclosure is directed to monomers and methods for synthesizing oligonucleotides comprising at least one nucleoside comprising a 3-hydroxyl group.

Modified Compounds and Uses Thereof

The present disclosure provides oligomeric compound comprising a modified oligonucleotide having a central region comprising one or more modifications. In certain embodiments, the present disclosure provides oligomeric compounds having an improved therapeutic index or an increased maximum tolerated dose.

Tuning CRISPR/Cas9 activity with chemically modified nucleotide substitutions

The present disclosure provides CRISPR/Cas9 ribonucleoprotein compositions comprising chemically modified CRISPR RNA (crRNA) guide and trans-acting CRISPR RNA (tracrRNA) components. Methods of using the disclosed CRISPR/Cas9 ribonucleoprotein compositions are also provided.