Patent classifications
C12N2330/10
SELF-REPLICATING CELL SELECTIVE GENE DELIVERY COMPOSITIONS, METHODS, AND USES THEREOF
Described herein are cell-selective mRNA constructs that can contain a RNA of interest and one or more miRNA targets. The cell-selective mRNA constructs described herein can be used to express an RNA of interest to a cell in a cell-selective manner.
SELF-REPLICATING CELL SELECTIVE GENE DELIVERY COMPOSITIONS, METHODS, AND USES THEREOF
Described herein are cell-selective mRNA constructs that can contain a RNA of interest and one or more miRNA targets. The cell-selective mRNA constructs described herein can be used to express an RNA of interest to a cell in a cell-selective manner.
COMPOSITIONS COMPRISING SMALL EXTRACELLULAR VESICLES DERIVED FROM UMBILICAL CORD BLOOD MONONUCLEAR CELLS WITH ANTI-INFLAMMATORY AND IMMUNOMODULATORY PROPERTIES AND PROCESS FOR OBTAINING THEM
The present invention relates to a process for isolating Small Extracellular Vesicles secreted by umbilical cord blood mononuclear cells (UCBMNCs) and compositions comprising said Small Extracellular Vesicles, which are useful to be applied to autoimmune diseases therapeutics or prophylactics and/or cosmetic purposes.
The proposed process for isolating UCBMNCs Small Extracellular Vesicles comprises three main steps: i) a first step of sequential centrifugation, ii) a second step of microfiltration combined with ultrafiltration (UF), and iii) a third step of size exclusion chromatography (SEC) and aims to achieve highly pure Small Extracellular Vesicles and in a higher yield.
The SEVs compositions comprise specific type of proteins, RNA and lipids, that enables them to be very effective when applied to inflammatory and autoimmune diseases therapeutics, such as psoriasis, lupus, atopic dermatitis, eczema, etc. and also to cosmetic or prophylactic compositions.
Therefore, the present invention lays in the technical domain of pharmaceuticals, medicine, cosmetics, research and development in cellular biology and appliances thereof.
TREATMENT OF GLIAL CELL DERIVED NEUROTROPHIC FACTOR (GDNF) RELATED DISEASES BY INHIBITION OF NATURAL ANTISENSE TRANSCRIPT TO GDNF
The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of Glial cell derived neurotrophic factor (GDNF), in particular, by targeting natural antisense polynucleotides of Glial cell derived neurotrophic factor (GDNF). The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of GDNF.
TREATMENT OF GLIAL CELL DERIVED NEUROTROPHIC FACTOR (GDNF) RELATED DISEASES BY INHIBITION OF NATURAL ANTISENSE TRANSCRIPT TO GDNF
The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of Glial cell derived neurotrophic factor (GDNF), in particular, by targeting natural antisense polynucleotides of Glial cell derived neurotrophic factor (GDNF). The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of GDNF.
Compositions and processes for targeted delivery, expression and modulation of coding ribonucleic acids in tissue
Compositions for expressing a polypeptide within a target organ, the composition comprising a delivery particle, and at least a first mRNA sequence complexed with, encapsulated by, or otherwise associated with the delivery particle, wherein the mRNA sequence comprises at least one coding sequence which codes for at least one polypeptide, at least a first untranslated region (UTR) sequence, and at least one micro-RNA (miRNA) binding site sequence, wherein the miRNA binding site sequence is located within, immediately 5′ to, or immediately 3′ to, the first UTR sequence; and wherein the miRNA binding site sequence provides for differential expression of the coding sequence between first and second cell types comprised within the target organ.
Compositions and processes for targeted delivery, expression and modulation of coding ribonucleic acids in tissue
Compositions for expressing a polypeptide within a target organ, the composition comprising a delivery particle, and at least a first mRNA sequence complexed with, encapsulated by, or otherwise associated with the delivery particle, wherein the mRNA sequence comprises at least one coding sequence which codes for at least one polypeptide, at least a first untranslated region (UTR) sequence, and at least one micro-RNA (miRNA) binding site sequence, wherein the miRNA binding site sequence is located within, immediately 5′ to, or immediately 3′ to, the first UTR sequence; and wherein the miRNA binding site sequence provides for differential expression of the coding sequence between first and second cell types comprised within the target organ.
High performance multi-input microRNA sensors and uses thereof
Provided herein are genetic circuits and cell state classifiers for detecting the microRNA profile of a cell. The cell state classifiers of the present disclosure are designed to incorporate multiple genetic circuits integrated together by transcriptional or translational control. Multiple inputs can be sensed simultaneously by coupling their detection to different portions of the genetic circuit such that the output molecule is produced only when the correct input profile of miRNAs is detected. The genetic circuits and cell state classifiers may be used in various applications (e.g., therapeutic or diagnostic applications).
High performance multi-input microRNA sensors and uses thereof
Provided herein are genetic circuits and cell state classifiers for detecting the microRNA profile of a cell. The cell state classifiers of the present disclosure are designed to incorporate multiple genetic circuits integrated together by transcriptional or translational control. Multiple inputs can be sensed simultaneously by coupling their detection to different portions of the genetic circuit such that the output molecule is produced only when the correct input profile of miRNAs is detected. The genetic circuits and cell state classifiers may be used in various applications (e.g., therapeutic or diagnostic applications).
SMALL RNA MEDICAMENT FOR PREVENTION AND TREATMENT OF INFLAMMATION-RELATED DISEASES AND COMBINATION THEREOF
Provided is a small RNA, a composition comprising the small RNA, a method for using same and use of same. The small RNA or composition can inhibit the ability of any one or more of pathways or genes listed in Table 3, or decrease or down regulate the expression level of IL-1 beta, IL-6, and/or TNF-alpha in vitro or in vivo, or treat or prevent IL-1 beta, IL-6, and/or TNF-alpha related diseases and/or increase cell viability in a subject.