The invention provides an oligonucleotide comprising an inosine, and/or a nucleotide containing a base able to form a wobble base pair or a functional equivalent thereof, wherein the oligonucleotide, or a functional equivalent thereof, comprises a sequence which is complementary to at least part of a dystrophin pre-m RNA exon or at least part of a non-exon region of a dystrophin pre-m RNA said part being a contiguous stretch comprising at least 8 nucleotides. The invention further provides the use of said oligonucleotide for preventing or treating DMD or BMD.

Provided herein are methods, compounds, and compositions for reducing expression of a nrRNA in an animal. Also provided herein are methods, compounds, and compositions for treating, ameliorating, delaying or reducing a symptom of a disease or disorder associated with a nuclear-retained RNA in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate a disease or condition associated with a nuclear-retained RNA, or a symptom thereof.

In one aspect, the invention relates to a method of loading exosomes with oligonucleotide cargo, by incubating an oligonucleotide comprising one or more hydrophobic modifications with a population of exosomes for a period of time sufficient to allow loading of the exosomes with the oligonucleotide. Exosomes loaded with hydrophobic ally modified oligonucleotide cargo, and uses thereof, are also provided.

The invention provides an artificial sgRNA and a CRISPR/Cas9 system by combining the artificial sgRNA and Cas9. Activity of the sgRNA can be retained even when a nucleotide linker region for forming a single strand by linking the 3-terminal of crRNA and the 5-terminal of tracrRNA in sgRNA is substituted with an amino acid derivative linker, when the linker region existing between stem-loop 1 and stem-loop 2 of tracrRNA and/or the loop portion of stem-loop 2 are/is substituted with an amino acid derivative linker, or when an amino acid derivative linker is added/inserted into the vicinity of the 5-terminal and/or the 3-terminal of sgRNA. Stability in vivo can be improved by introducing one or more amino acid derivative linkers into the sgRNA.

Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of ATXN3 mRNA in a cell or animal, and in certain instances reducing the amount of Ataxin-3 protein in a cell or animal. Such compounds, methods, and pharmaceutical compositions are useful to prevent or ameliorate at least one symptom or hallmark of a neurodegenerative disease. Such symptoms and hallmarks include ataxia, neuropathy, and aggregate formation. Such neurodegenerative diseases include SCA3.

CRISPNA, a new tool for genome editing and diagnosis. The present invention relates to methods and systems for genome editing and diagnosis, and specifically relates to use of Peptide Nucleic Acids (PNAs) to direct the Cas proteins to their DNA or RNA targets.

The present disclosure relates to the technical field of genetic engineering, in particular to a target ligand. The target ligand provided by the present disclosure forms a siRNA conjugate with a specific small interfering RNA sequence, which targets to an angiopoietin like 3 (ANGPTL3), and reduce the expression quantity of an ANGPTL3 protein by degrading a transcript of an ANGPTL3 gene in a cell. Therefore, the siRNA conjugate formed by the target ligand provided by the present disclosure may be used to prevent and/or treat a dyslipidemia disease.

The present disclosure relates to antisense oligomers and related compositions and methods for increasing the expression of functional human type VII collagen and methods for treating dystrophic epidermolysis bullosa and related disorders and relates to inducing exclusion of exon 80 in human type VII collagen mRNA.

The present disclosure provides systems and methods that can provide portable, real-time accessible DNA memories. An example DNA-based data storage system includes a loading region configured to receive a plurality of DNA-based data storage elements in a suspension fluid and a plurality of microtubes disposed in a capture/release region. The microtubes are configured to capture and release the DNA-based data storage elements. The DNA-based data storage system also includes a linearization region configured to linearize the DNA-based data storage elements and a readout region with a readout device configured to provide information indicative of the respective DNA-based data storage elements.

The present invention relates to a pharmaceutical composition that is for treating pancreatic cancer and contains a nucleic acid complex as an active ingredient, and more specifically, to a pharmaceutical composition that is for preventing or treating pancreatic cancer and contains a nucleic acid complex in which a bioactive peptide nucleic acid, having a sequence binding to the KRAS gene, and a carrier peptide nucleic acid are complementarily bound. The nucleic acid complex according to the present invention has excellent cell permeability and intracellular activity, and can effectively inhibit the expression of the KRAS gene and subtype genes thereof, and is thus useful for the prevention or treatment of pancreatic cancer.