Abstract: This disclosure relates to the synthesis of novel modified oligonucleotides. The synthesis of novel phosphoramidites are also provided.

Abstract: This disclosure provides compositions, systems, and methods for the delivery of therapeutic oligonucleotides to the lung. The oligonucleotide is conjugated to a dendron comprising a hydrophilic end group, a phosphate group, and/or a hydrophobic chain.

Abstract: This disclosure provides compositions, systems, and methods for the delivery of therapeutic oligonucleotides to the brain. The oligonucleotide is conjugated to a dendron comprising a hydrophilic end group, a phosphate group, and/or a hydrophobic chain.

Abstract: The present invention is related to the treatment of muscular dystrophy. In particular, compositions of short interfering ribonucleic acids (siRNAs) Eire contemplated that are targeted to different portions of a DUX4 messenger ribonucleic acid molecule. Preferably, these siRNAs are double stranded having a sense strand of fifteen (15) nucleotides. When applied as a therapeutic method, the short interfering ribonucleic acids reduce DUX4 protein translation and mediate a therapeutic response in human facioscapulohumeral muscular dystrophies, FSHD 1 and FSHD2.

Abstract: This disclosure provides compositions, systems, and methods for the delivery of therapeutic oligonucleotides to the skin. The oligonucleotide is conjugated to a dendron comprising a hydrophilic end group, a phosphate group, and/or a hydrophobic chain.

Abstract: This disclosure relates to the synthesis of novel modified oligonucleotides. The synthesis of novel phosphoramidites are also provided.

Abstract: Novel oligonucleotides that enhance silencing of the expression of a gene containing a single nucleotide polymorphism (SNP) relative to the expression of the corresponding wild-type gene are provided. Methods of using novel oligonucleotides that enhance silencing of the expression of a gene containing a SNP relative to the expression of the corresponding wild-type gene are provided.

Abstract: This disclosure provides compositions, systems, and methods for the topical delivery of therapeutic oligonucleotides with microneedle particles.

Abstract: Provided herein are stable, modified oligonucleotides comprising a 5?-alkyl phosphate or derivatives thereof and methods for preparing the same.

Abstract: This disclosure relates to novel HTT-1A targeting sequences. Novel HTT-1A targeting oligonucleotides for the treatment of neurodegenerative diseases are also provided.

Abstract: This disclosure provides compositions, systems, and methods for the delivery of therapeutic oligonucleotides. The oligonucleotide is conjugated to a functional moiety comprising a carbohydrate. In certain embodiments, the carbohydrate is a glucosamine or a derivative thereof.

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

Abstract: Provided herein are self-delivering oligonucleotides that are characterized by efficient RISC entry, minimum immune response and off-target effects, efficient cellular uptake without formulation, and efficient and specific tissue distribution.

Abstract: This disclosure relates to novel SARS-CoV-2 targeting sequences. Novel SARS-CoV-2 targeting oligonucleotides for the treatment of SARS-CoV-2 infection are also provided.

Abstract: This disclosure relates to novel IFN-? signaling pathway target gene targeting sequences. Novel IFNGR1, JAK1, JAK2, and STAT1 targeting oligonucleotides for the treatment of vitiligo are also provided.

Abstract: Provided are compositions and methods comprising two-tailed siRNAs (tt-siRNAs) that exhibit unprecedented cellular uptake and silencing. Also provided are methods of treating neurological and other diseases with the two-tailed siRNAs of the invention.

Abstract: This disclosure relates to a therapeutic combination of drugs for the treatment or management of a neurodegenerative disease, the combination comprising: a first conjugate comprising an RNA silencing agent and a first targeting agent that targets the first conjugate to the central nervous system, and a second conjugate comprising an antagonist of the RNA silencing agent and a second targeting agent that targets the second conjugate to a off-target tissue.

Abstract: Novel oligonucleotides that enhance silencing of the expression of a gene containing a single nucleotide polymorphism (SNP) relative to the expression of the corresponding wild-type gene are provided. Methods of using novel oligonucleotides that enhance silencing of the expression of a gene containing a SNP relative to the expression of the corresponding wild-type gene are provided.

Abstract: Novel artificial exosomes and methods for producing novel artificial exosomes are provided. Methods of delivering cargo molecules to a cell using artificial exosomes are also provided.

Abstract: Provided herein are oligonucleotides (e.g., siRNA) targeting S6K1. Also provided are methods of treating a disease associated with S6K1 expression.