C07K2319/07

CELL MEMBRANE PENETRATING CONJUGATES FOR GENE EDITING
20220056483 · 2022-02-24 ·

A genome-editing complex for modifying a target polynucleotide comprising a recombinant β helical protein linked to either one or more molecules of a genome-editing system or a plasmid encoding for one or more molecules of a genome-editing system, wherein the β helical protein length is in the range of from 5 nm to 25 nm, and width is in the range of from 1 nm to 5 nm.

CONJUGATES FOR DELIVERY TO MITOCHONDRIA
20170247674 · 2017-08-31 ·

The present disclosure relates generally to conjugates and their uses and production. More particularly, it concerns conjugates comprising an A44 or K39 gingipain adhesion peptide, or fragment thereof (e.g., an A44/K39 peptide portion) covalently attached to a heterologous agent that is capable of entering a cell by endocytosis and translocating to mitochondria.

METHODS AND COMPOSITIONS FOR TREATMENT OF MITOCHONDRIAL DISORDERS

The present invention concerns in general novel fusion proteins comprising a membrane transferring moiety and an enzymatic moiety. The present invention further concerns a method of treating disease using said fusion proteins.

REDIRECTING DEATH-INDUCING SIGNAL COMPLEX (DISC) BY MODIFYING DEATH RECEPTOR AGONIST TO INDUCE CELL DEATH FOR CANCER TREATMENT
20220306768 · 2022-09-29 · ·

It was found that TRAIL-induced death signaling was largely diminished by lysosomal degradation system. Inhibition of lysosomal degradation by small molecules led to accumulation of DR5 in lysosomes and reversed TRAIL resistance in almost all cancer cells. Redirecting TRAIL-induced signaling away from lysosomal degradation may therefore induce massive cell death and overcome TRAIL resistance. Taking advantage of bioactive protein transduction domains (PTD) and signaling peptides, such as TAT peptide from HIV TAT protein, NLS (nuclear localization signal), MTS (mitochondrial targeting sequence), a series of new TRAILs fused with these peptides was constructed. It was found that TRAIL fused with bioactive peptide exerted remarkably high potency in inducing cell death in cancer cells, but not normal cells.

PH-responsive proteolysis probe
11203621 · 2021-12-21 · ·

The present invention provides a tool which exhibits excellent properties in the quantification of autophagy activity. A unimolecular FRET probe of the present invention includes an acceptor consisting of a fluorescent protein to be enzymatically degraded inside a lysosome or a vacuole; and a donor having an amino acid sequence having a sequence identity of 95% or more with respect to an amino acid sequence represented by SEQ ID NO: 1.

Method for the generation of compact tale-nucleases and uses thereof

The present invention relates to a method for the generation of compact Transcription Activator-Like Effector Nucleases (TALENS) that can efficiently target and process double-stranded DNA. More specifically, the present invention concerns a method for the creation of TALENs that consist of a single TALE DNA binding domain fused to at least one catalytic domain such that the active entity is composed of a single polypeptide chain for simple and efficient vectorization and does not require dimerization to target a specific single double-stranded DNA target sequence of interest and process DNA nearby the DNA target sequence. The present invention also relates to compact TALENs, vectors, compositions and kits used to implement the method.

DNA BINDING PROTEINS AND USES THEREOF
20210380648 · 2021-12-09 ·

The present disclosure relates to compositions and methods for modulating gene expression and in particular to DNA binding proteins and their use for increasing expression of Klotho. In some examples, the present disclosure provides an isolated or recombinant DNA binding protein comprising or attached to a transcriptional activation domain wherein the DNA binding protein binds to a target sequence within or near a Klotho gene.

Compositions and methods for treating leber's hereditary optic neuropathy with NADH dehydrogenase proteins

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.

Compositions and methods for treating Leber's hereditary optic neuropathy

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.

Expression of Nitrogenase Polypeptides In Plant Cells

The present invention relates to methods and means for producing nitrogenase polypeptides in the mitochondria of plant cells.