A system for producing a therapeutic oligomer includes a computing device configured to design a proposed therapeutic oligomer sequence, wherein designing further comprises generating a genomic library for an organism from a gene target, initiating a sequence identification function, identifying a genomic locus that the proposed therapeutic oligomer sequence is predicted to bond to as a function of an off-target sequence function, selecting the proposed therapeutic oligomer sequence as a function of the sequence identification function, the genomic locus, and a criterion element, and synthesize a therapeutic oligomer as a function of the proposed therapeutic oligomer sequence.

The present disclosure relates to compounds useful for the detection or modulation of target nucleic acids, including DNA and RNA. The present disclosure further relates to methods for treatment of trinucleotide repeat disorders, which can include administration of oligonucleotide analogues that can bind pathogenic nucleotide repeats in DNA or RNA.

The present invention relates to a composition for preventing or treating macular degeneration, containing a cell permeable nucleic acid complex as an active ingredient and, more specifically, to a pharmaceutical composition for preventing, ameliorating, or treating macular degeneration comprising, a cell-permeable nucleic acid complex containing a bioactive nucleic acid targeting an NLRP3 gene; and a carrier peptide nucleic acid which are complementarily bound to said bioactive nucleic acid, as an active ingredient. The cell permeable nucleic acid complex, in which the bioactive nucleic acid targeting the NLRP3 gene and the carrier peptide nucleic acid are complementarily bound to each other, according to the present invention, has high cell permeability and thus inhibits the expression of NLRP3 without direct injection, and thereby is useful for preventing, ameliorating, or treating macular degeneration.

Antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon 53 skipping are described.

Provided are peptide nucleic acid derivatives targeting a 3′ splice site of the human tyrosinase pre-mRNA. The peptide nucleic acid derivatives potently induce a splice variant of the human tyrosinase mRNA in cells, and are useful to safely treat dermatological indications or conditions involving the human tyrosinase protein upon topical administration.

The present disclosure features useful compositions and methods to treat disorders for which deamination of an adenosine in an mRNA produces a therapeutic result, e.g., in a subject in need thereof.

Antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon 53 skipping are described.

A nucleic acid complex having a novel structure, which may introduce a bioactive nucleic acid into cells, a composition for treating or diagnosing disease including the same, and a method of regulating target gene expression using the same, are described. The nucleic acid complex may include a bioactive nucleic acid, which includes a material for facilitating endosomal escape, and a carrier peptide nucleic acid, complementarily bound to each other, as useful in a composition for treating or diagnosing disease, or a composition for regulating target gene expression. Such nucleic acid complex may increase the stability of the bioactive nucleic acid, reduce loss of the bioactive nucleic acid, such as precipitation caused by self-aggregation, increase the intracellular delivery efficiency of the bioactive nucleic acid, and easily regulate target gene expression.

The invention provides compositions and methods of making and using effector oligonucleotides, including effector oligonucleotides with greater than one mismatch as compared to its target sequence. These effector oligonucleotides are useful for improving the efficiency of genomic editing as well as providing therapeutic benefits to individuals in need thereof.

Provided herein are methods, compounds, and compositions for reducing expression of a DMPK mRNA and protein in an animal. Also provided herein are methods, compounds, and compositions for preferentially reducing CUGexp DMPK RNA, reducing myotonia or reducing spliceopathy in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate type 1 myotonic dystrophy, or a symptom thereof.