C07K2319/07

COMPOSITIONS AND METHODS FOR TREATING LEBER'S HEREDITARY OPTIC NEUROPATHY
20220340895 · 2022-10-27 ·

Disclosed herein is a recombinant nucleic acid, comprising: a mitochondrial targeting sequence; a mitochondrial protein coding sequence, wherein said mitochondrial protein coding sequence encodes a polypeptide comprising a mitochondrial protein; and a 3′UTR nucleic acid sequence. Also disclosed is a pharmaceutical composition comprising the recombinant nucleic acid and a method of treating Leber's hereditary optic neuropathy (LHON) using the pharmaceutical composition.

Mitochondrial Targeted Releasable Linker
20220273664 · 2022-09-01 ·

There is described herein compound comprising a mitochondrial targeting portion, a cargo portion including a drug unit, and a linker conjugating the mitochondrial targeting portion and the cargo portion, the linker portion cleavable in a mitochondrion of a cell for preferentially releasing the cargo portion within the mitochondrion as compared to a cytoplasm of the cell.

Materials and methods for treating Friedreich's Ataxia

A Transactivator of Transcription-frataxin (TAT-FXN) fusion polypeptide useful in treating subjects diagnosed with Friedreich's Ataxia, hypertrophic cardiomyopathy, or both are disclosed, as are related methods of treatment and pharmaceutical compositions.

COMPOSITIONS AND METHODS FOR TREATING LEBER'S HEREDITARY OPTIC NEUROPATHY
20220259619 · 2022-08-18 ·

Disclosed herein is a recombinant nucleic acid, comprising: a mitochondrial targeting sequence; a mitochondrial protein coding sequence, wherein said mitochondrial protein coding sequence encodes a polypeptide comprising a mitochondrial protein; and a 3′UTR nucleic acid sequence. Also disclosed is a pharmaceutical composition comprising the recombinant nucleic acid and a method of treating Leber's hereditary optic neuropathy (LHON) using the pharmaceutical composition.

COMPOSITION COMPRISING MLS-STAT3 FOR PREVENTION OR TREATMENT OF IMMUNE DISEASE

The present invention relates to: a recombinant peptide in which a Mitochondria Localization Sequence (MLS) peptide and the Signal Transducer and Activator of Transcription 3 (STAT3) are fused to each other; a recombinant vector carrying a polynucleotide coding for the recombinant peptide; and a composition comprising the recombinant peptide or the recombinant vector as an active ingredient for prevention or treatment of autoimmune disease or inflammatory disease, wherein the recombinant peptide or the recombinant vector allows STAT3 to be overexpressed in the mitochondria to enhance the mitochondrial function, resulting in inhibiting the expression of inflammatory cytokines including IL-17, whereby the composition may be advantageously used for preventing or treating autoimmune disease or inflammatory diseases.

Compositions and methods for treating SARS-CoV-2 infections

The present application relates to methods and compositions and methods for treating viral infections, especially those caused by SARS-CoV-2. In one aspect, a method of treatment comprises administering to a subject in need of such treatment an effective amount of a pharmaceutical composition comprising a multipartite SARS-CoV-2-inhibiting peptide comprising a secretion modulating region (VI-SMR) peptide from HIV-1 Nef in combination with a cell-penetrating peptide (CPP) domain, a Clusterin (Clu)-binding peptide (Clu-BP) domain, a mitochondrial targeting (Mito-T) peptide domain, an anti-fusogenic (AF) peptide domain, a viral attachment inhibitor (VAI) domain or combination thereof, optionally where the SARS-CoV-2-inhibiting peptide is pegylated and/or modified with one or more hydrophobic domains.

COMPOSITIONS AND METHODS FOR TREATING LEBER'S HEREDITARY OPTIC NEUROPATHY WITH NADH DEHYDROGENASE PROTEINS
20220273816 · 2022-09-01 ·

Disclosed herein is a recombinant nucleic acid, comprising: a mitochondrial targeting sequence; a mitochondrial protein coding sequence, wherein said mitochondrial protein coding sequence encodes a polypeptide comprising a mitochondrial protein; and a 3′UTR nucleic acid sequence. Also disclosed is a pharmaceutical composition comprising the recombinant nucleic acid and a method of treating Leber's hereditary optic neuropathy (LHON) using the pharmaceutical composition.

GENETICALLY ENGINEERED PLANTS THAT EXPRESS A QUINONE-UTILIZING MALATE DEHYDROGENASE

Genetically engineered plants that express a quinone-utilizing malate dehydrogenase (MQO) are provided. The plant comprises a modified gene for the quinone-utilizing malate dehydrogenase. The modified gene comprises (i) a promoter and (ii) a nucleic acid sequence encoding the quinone-utilizing malate dehydrogenase. The promoter is non-cognate with respect to the nucleic acid sequence encoding the quinone-utilizing malate dehydrogenase. The modified gene is configured such that transcription of the nucleic acid sequence is initiated from the promoter and results in expression of the quinone-utilizing malate dehydrogenase. The plants can express the quinone-utilizing malate dehydrogenase in mitochondria of cells of the plants. Conversion of malate to oxaloacetate in the mitochondria can be increased, resulting in increased crop performance and/or seed, fruit or tuber yield. Methods and compositions for making the plants also are provided.

Antibody mimetic capable of being activated reversibly and uses thereof

The present invention relates to the preparation of an antibody analogue capable of being activated reversibly, and uses thereof, and provides a fusion protein comprising an inactive first fragment of an antibody analogue is fused to a stimulus-induced dimerization protein.

IMPORTATION OF MITOCHONDRIAL PROTEIN BY AN ENHANCED ALLOTOPIC APPROACH

An expression vector containing appropriate mitochondrion-targeting sequences (MTS) and appropriate 3′UTR sequences provides efficient and stable delivery of a mRNA encoding a protein (CDS) to the mitochondrion of a mammalian cell. The MTS and 3′UTR sequences guide the CDS mRNA from the nuclear compartment of the cell to mitochondrion-bound polysomes, where the CDS is translated. This provides an efficient translocation of a mature functional protein into the mitochondria. A method of targeting mRNA expressed in the nuclear compartment of a mammalian cell to the mitochondrion is also provided. The vector and methods can be used to treat defects in mitochondrial function.