Peptide nucleic acid (PNA) oligomers that target the β-globin gene and can increase the frequency of recombination of donor oligonucleotide at the site of a Sickle Cell Disease mutation are provided. Nanoparticle formulations for delivering the PNA oligomers and donor oligonucleotides, and potentiating agents for increase the frequency of recombination of the donor oligonucleotide are also provided. Methods of using the PNA oligomers, donor oligonucleotides, nanoparticles, and potentiating agents for treating Sickle Cell Disease are also provided.

A chimeric molecule resulting from fusion of a first nucleic acid or a derivative thereof, which has an ability to bind to a target nucleic acid, with a second nucleic acid or a derivative thereof, which has an ability to bind to the target nucleic acid, and in which a main chain skeleton. is anionic, a pharmaceutical composition containing the chimeric molecule, a method for cleaving a target nucleic acid using the chimeric molecule, and a kit for target nucleic acid cleavage or diagnosis including the chimeric molecule.

A TLR7/8 inhibitor for reduction of bone resorption, especially in chronic joint diseases, and to a pharmaceutical composition including the inhibitor. A method for predicting the severity of the course of disease of rheumatoid arthritis in a patient.

Disclosed are methods for modulating splicing of Tau mRNA in an animal with Tau antisense compounds. Also disclosed herein are methods for reducing expression of Tau mRNA and protein in an animal with Tau antisense compounds. Such compounds and methods are useful to treat, prevent, or ameliorate neurodegenerative diseases in an individual in need thereof. Examples of neurodegenerative diseases that can be treated, prevented, and ameliorated with the administration Tau antisense oligonucleotides include Alzheimer's Disease, Fronto-temporal Dementia (FTD), FTDP-17, Progressive Supranuclear Palsy, Chronic Traumatic Encephalopathy, Epilepsy, and Dravet's Syndrome.

The present invention relates to antisense oligonucleotides and/or compounds that modulate the expression of and/or function of Filaggrin (FLG), in particular, by targeting natural antisense polynucleotides of Filaggrin (FLG). The invention also relates to the identification of these antisense oligonucleotides and/or compounds and their use in treating diseases and disorders associated with the expression of FLG.

Disclosed herein include methods and compositions for selectively amplifying and/or extending nucleic acid target molecules in a sample. The methods and compositions can, for example, reduce the amplification and/or extension of undesirable nucleic acid species in the sample, and/or allow selective removal of undesirable nucleic acid species in the sample.

Triplex-forming peptide nucleic acid (PNA) oligomers having a γ-substitution in one or more residues of the Hoosteen binding segment are provided. γPNA-containing triplex-forming molecules can be used in combination with a donor DNA fragment to facilitate genome modification in vitro and in vivo. In some embodiments, the oligomers have between 1 and 50 inclusive γ-substituted PNA residues.

Provided herein are single-stranded tile (SST) structures prepared from and comprising single-stranded γPNA (ss-γPNA) strands, along with methods of making an SST structure from single-stranded γPNA (ss-γPNA) strands.

The invention relates to improved ANTISENSE agents for the treatment of gram-negative bacterial infections. Compounds of the invention utilise an Antibiotic-Assisted Translocation; AAT′ platform to improve influx into bacterial cells through enhanced permeability, providing improved intracellular exposure of the ANTISENSE AGENT and superior treatment of the infection.

Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of PMP22 RNA in a cell or animal, and in certain instances reducing the amount of PMP22 protein in a cell or animal. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease. Such symptoms and hallmarks include demyelination, progressive axonal damage and/or loss, weakness and wasting of foot and lower leg muscles, foot deformities, and weakness and atrophy in the hands. Such neurodegenerative diseases include Charcot-Marie-Tooth disease.