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.

The present disclosure relates to polypeptides, such as fibronectin type III (FN3) domains that can bind CD71, their conjugates, isolated nucleotides encoding the molecules, vectors, host-cells, as well as methods of making and using the same.

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

The present invention relates to antisense oligonucleotides that are complimentary to SOD1, leading to decreased expression of SOD1. Reduced expression of SOD1 is beneficial in medical disorders such as Amyotrophic Lateral Sclerosis.

Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload inhibits expression or activity of a DMPK allele comprising a disease-associated-repeat. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide or RNAi oligonucleotide.

The present invention provides an immunity-inducing agent comprising a polynucleotide-peptide conjugate or a pharmaceutically acceptable salt thereof as an active component, wherein the polynucleotide-peptide conjugate consists of: a single-chain polynucleotide or polynucleotide derivative comprising a CpG motif; a peptide; and a spacer which is covalently bonded at one end thereof to the polynucleotide or polynucleotide derivative and covalently bonded at the other end thereof to the peptide, wherein the peptide is a peptide modified by substituting one or more contiguous amino acids at the N-terminus of an MHC-binding peptide with an amino acid having a reactive functional group which allows for the formation of a covalent bond with the spacer, wherein the one or more contiguous amino acids contain no anchor residues for MHC binding.

The present invention provides methods for cancers associated with a TERT promoter mutation in a subject. In some embodiments, the methods comprise administering to the subject a therapeutically effective amount of an agent that specifically reduces or inhibits GA binding protein transcription factor beta subunit 1 long isoform (GABPBIL) expression or function.

The present invention is directed to an aptamer composition comprising at least one oligonucleotide consisting of: deoxyribonucleotides, ribonucleotides, derivatives of deoxyribonucleotides, derivatives of ribonucleotides, and mixtures thereof; wherein said aptamer composition has a binding affinity for one or more bacterial species from the genera Prevotella and Porphyromonas.

Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload inhibits expression or activity of DUX4. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide or RNAi oligonucleotide.

The present disclosure provides nucleic acid carriers comprising targeting agents, pharmaceutical compositions comprising the same, and methods of making and using the same.