C07K14/003

HIF 1-alpha antisense oligonucleotides

Provided are peptide nucleic acid derivatives targeting a part of the human HIF-1α pre-mRNA. The peptide nucleic acid derivatives potently induce exon skipping to yield splice variants of HIF-1α mRNA in cells, and are useful to treat indications or conditions involving the overexpression of HIF-1α.

Nucleic acid-polypeptide compositions and methods of inducing exon skipping

Disclosed herein are molecules and pharmaceutical compositions that induce an insertion, deletion, duplication, or alteration in an incorrectly spliced mRNA transcript to induce exon skipping or exon inclusion. Also described herein include methods for treating a disease or disorder that comprises a molecule or a pharmaceutical composition that induces an insertion, deletion, duplication, or alteration in an incorrectly spliced mRNA transcript to induce exon skipping or exon inclusion.

THYCLOTIDES

Disclosed are thyclotides, which are oligomers, each comprising (a) from about 8 to about 25 monomer units of formula (I) and (b) from 0 to about 24 monomer units of formula (II): wherein B is a nucleobase, which can be the same or different at each occurrence, or a pharmaceutically acceptable salt thereof. The thyclotides are soluble in water, bind strongly to complementary DNA and RNA, and are cell permeable. The thyclotides are useful as reagents for antisense and antigene applications, and as probes in molecular diagnostics and microarrays.

METHODS AND COMPOSITIONS FOR TREATING SARS-COV-2 INFECTION USING PEPTIDE NUCLEIC ACID-BASED AGENTS
20230242588 · 2023-08-03 ·

The invention relates generally to viral infections and more specifically to compositions and methods for treating infections by SARS-CoV-2 (2019-nCoV). In particular, the invention provides a PNA agent which includes a PNA moiety with a sequence that targets a SARS-CoV-2 gene; a first cationic and hydrophobic peptide at the N-terminus of the PNA moiety, wherein the first peptide comprises lysine residues, and at least one of the lysine residues comprises a palmitoyl side chain moiety; and a second cationic and hydrophobic peptide at the C-terminus of the PNA moiety, wherein the second peptide comprises lysine residues, and at least one of the lysine residues comprises a palmitoyl side chain moiety.

ACETYL-COA CARBOXYLASE2 ANTISENSE OLIGONUCLEOTIDES

The present invention provides the peptide nucleic acid derivative which targets 5′ splice site of the human ACC2 pre-mRNA “exon 12”. The peptide nucleic acid derivatives in the present invention strongly induce splice variants of the human ACC2 mRNA in cell and are very useful to treat conditions or disorders of skin aging associated with the human ACC2 protein.

METHODS AND COMPOSITIONS FOR PROTEIN SEQUENCING
20210364527 · 2021-11-25 · ·

Aspects of the application provide methods of identifying and sequencing proteins, polypeptides, and amino acids, and compositions useful for the same. In some aspects, the application provides amino acid recognition molecules, such as amino acid binding proteins and fusion polypeptides thereof. In some aspects, the application provides amino acid recognition molecules comprising a shielding element that enhances photostability in polypeptide sequencing reactions.

Nucleic acid-polypeptide compositions and methods of inducing exon skipping

Disclosed herein are molecules and pharmaceutical compositions that induce an insertion, deletion, duplication, or alteration in an incorrectly spliced mRNA transcript to induce exon skipping or exon inclusion. Also described herein include methods for treating a disease or disorder that comprises a molecule or a pharmaceutical composition that induces an insertion, deletion, duplication, or alteration in an incorrectly spliced mRNA transcript to induce exon skipping or exon inclusion.

PREPARATION OF OLIGO CONJUGATES

Conjugated molecules are prepared that comprise a predetermined number of oligo conjugation components. The conjugated molecules also may comprise one or more detectable labels. Preparation of these molecules can be implemented according to an asymmetric or a symmetric conjugation strategy.

PEPTIDE-NUCLEIC ACID COMPLEX
20220010032 · 2022-01-13 ·

There is provided a method for producing a peptide-nucleic acid complex containing a peptide and a nucleic acid encoding the peptide. The method for producing a peptide-nucleic acid complex includes a step of preparing a nucleic acid to which a transpeptidase N-terminal substrate motif has been added, the nucleic acid containing a first coding sequence encoding a peptide, a second coding sequence encoding a transpeptidase, and a third coding sequence encoding a transpeptidase recognition motif; a step of synthesizing a chimeric protein containing a domain of the peptide, a domain of the transpeptidase, and the transpeptidase recognition motif, from the nucleic acid to which the transpeptidase N-terminal substrate motif has been added, using a cell-free protein synthesis system; and a step of forming the peptide-nucleic acid complex by means of the transpeptidase domain.

METHODS AND COMPOSITIONS COMPRISING THERAPEUTIC GAMMA PEPTIDE NUCLEIC ACID-BASED MOLECULES

Disclosed are compounds and methods for the treatment of diseases or disorders associated with expression of miR-122. More specifically, γ peptide nucleic acid (γPNA) inhibitors of miR-122 and methods of using the same are disclosed.