C12N2310/52

PRNA three-way junctions

Three-way junction (3WJ) RNA scaffolds derived from phi29, M2, SF5, and GA1 pRNAs and which have high stability are described. The pRNA 3WJ scaffolds can be used to form compounds, conjugates, compositions, and nanoparticles for delivery of active agents for therapeutic and/or diagnostic functions.

OLIGONUCLEOTIDE-BASED MODULATION OF C9orf72

This disclosure relates to novel C9ORF72 targeting sequences. Novel oligonucleotides for the treatment of neurodegenerative diseases are also provided.

OLIGONUCLEOTIDES FOR TISSUE SPECIFIC APOE MODULATION
20200362341 · 2020-11-19 ·

This disclosure relates to novel ApoE targeting sequences. Novel oligonucleotides for the treatment of neurodegenerative and amyloid-related diseases are also provided.

RNA nanoparticles and method of use thereof

The presently-disclosed subject matter relates to an artificial RNA nanostructure and method of use thereof. In particular, the presently-disclosed subject matter relates to RNA nanoparticles and RNA dendrimers, and methods of disease diagnosis and treatments using the RNA nanostructure and RNA dendrimers.

Branched oligonucleotides

Provided herein are branched oligonucleotides exhibiting efficient and specific tissue distribution, cellular uptake, minimum immune response and off-target effects, without formulation.

OLIGONUCLEOTIDE COMPOUNDS FOR TARGETING HUNTINGTIN MRNA
20200308584 · 2020-10-01 ·

This disclosure relates to novel huntingtin targets. Novel oligonucleotides for the treatment of Huntington's disease are also provided.

RNA nanoparticle for treatment of gastric cancer

The presently-disclosed subject matter relates to RNA-based composition and method to treat gastric cancer in a subject. More particularly, the presently disclosed subject matter relates to a RNA nanostructure and composition containing a multiple branched RNA nanoparticle, a gastric cancer targeting module, and an effective amount of a therapeutic agent. Further, the presently disclosed subject matter relates to a method of using the RNA nanoparticle composition to treat gastric cancer in a subject having or at risk of having gastric cancer.

Oligonucleotide compounds for targeting huntingtin mRNA

This disclosure relates to novel huntingtin targets. Novel oligonucleotides for the treatment of Huntington's disease are also provided.

META-STABLE OLIGONUCLEOTIDES JUNCTIONS FOR DELIVERY OF THERAPEUTICS

Disclosed herein are multi-way oligonucleotide junctions for delivering one or more cargo molecules to a biological target and method of making such junctions. The oligonucleotide junctions are formed by two or more oligonucleotides and are stable outside the cell and easily dissociate inside the cell to release the cargo molecule(s). One or more cargo molecules as well as delivery ligand can be loaded to the junctions for targeted delivery. Also disclosed are nanostructures including one or more junctions attached to each other for delivering two or more cargo molecules.

PEPTIDE NUCLEIC ACID CONJUGATES
20200095577 · 2020-03-26 ·

The disclosure is directed to conjugates, e.g. PNA conjugates, as well as methods of employing the conjugates for detecting one or more targets in a biological sample, e.g. a tissue sample.