C07K14/003

Peptide nucleic acid conjugates
11597925 · 2023-03-07 · ·

The disclosure is directed to conjugates, e.g. PNA conjugates, as well as methods of employing the conjugates for detecting one or more targets in a biological sample, e.g. a tissue sample.

Modified peptide nucleic acid compositions

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.

METHOD FOR PRODUCING PNA OLIGOMER IN SOLUTION PROCESS

The present invention provides a method for producing a PNA oligomer. More specifically, the method comprises a step for reacting a PNA block and a PNA block in a solution to produce a PNA oligomer, and thus a PNA oligomer of interest can be produced with remarkably improved purity and yield.

TYROSINASE ANTISENSE OLIGONUCLEOTIDES

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.

PEPTIDE NUCLEIC ACID COMPLEX HAVING ENDOSOMAL ESCAPE CAPACITY, AND USE THEREOF
20230193275 · 2023-06-22 ·

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.

COMPLEXES FOR GENE DELETION AND EDITING
20230183752 · 2023-06-15 ·

Complexes comprising a nucleic acid-guided endonuclease, a sequence-specific targeting nucleic acid and an amphipathic helical peptide are provided. Compositions and methods for delivery of complexes comprising a nucleic acid-guided endonuclease, a sequence-specific targeting nucleic acid and an amphipathic helical peptide to mammals for both research and therapeutic use are provided. Methods of treating or reducing one or more symptoms of type 2 diabetes, prediabetes and/or gestational diabetes are provided.

PEPTIDE NUCLEIC ACID CONJUGATES
20230174585 · 2023-06-08 ·

The present disclosure is directed to conjugates of a specific binding entity and an oligomer, i.e. [Specific Binding Entity]-[Oligomer].sub.n, wherein n is an integer ranging from 1 to 12, and where the Oligomer includes, in some embodiments, a PNA sequence having at least one substituent at a gamma carbon position. In some embodiments, the substituent at the gamma carbon position, e.g. an amino acid, a peptide, a miniPEG, or a polymer, includes at least one reporter moiety.

MODIFIED PEPTIDE NUCLEIC ACID COMPOSITIONS

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.

COMPOSITIONS AND METHODS OF MAKING POLYMERIZING NUCLEIC ACIDS
20170327633 · 2017-11-16 ·

Provided herein are compositions and methods of making high density nucleic acid polymers.

SNAP25 antisense oligonucleotides

Provided are peptide nucleic acid derivatives targeting a 3′ splice site of the human SNAP25 pre-mRNA. The peptide nucleic acid derivatives potently induce at least a splice variant of the human SNAP25 mRNA in cells, and are useful to safely treat dermatological indications or conditions involving the expression of the human SNAP25 protein by topical administration.