A61K38/45

NEW USE OF CMTR1 HAVING SIRNA PRODUCTION AND FUNCTION ENHANCING ACTIVITY

The present disclosure relates to a novel use of CMTR1 having the activity of enhancing the production and function of siRNA, and more particularly, to a composition for enhancing the production of siRNA including a cap1 2′-O-ribose methyltransferase (CMTR1) protein as an active ingredient, and a composition for enhancing the gene silencing activity by siRNA, and also to a method for producing siRNA for gene silencing in vitro. According to the present disclosure, the CMTR1 may enhance the production of siRNA for gene silencing and at the same time, ultimately enhance the production and function of siRNA without artificial chemical modification of siRNA by enhancing the formation of holo-RNA-induced silencing complex (RISC) that acts on silencing of a target gene in an RNAi mechanism. Therefore, the CMTR1 of the present disclosure can be usefully used in the development of pharmaceuticals using siRNA as a therapeutic agent.

NEW USE OF CMTR1 HAVING SIRNA PRODUCTION AND FUNCTION ENHANCING ACTIVITY

The present disclosure relates to a novel use of CMTR1 having the activity of enhancing the production and function of siRNA, and more particularly, to a composition for enhancing the production of siRNA including a cap1 2′-O-ribose methyltransferase (CMTR1) protein as an active ingredient, and a composition for enhancing the gene silencing activity by siRNA, and also to a method for producing siRNA for gene silencing in vitro. According to the present disclosure, the CMTR1 may enhance the production of siRNA for gene silencing and at the same time, ultimately enhance the production and function of siRNA without artificial chemical modification of siRNA by enhancing the formation of holo-RNA-induced silencing complex (RISC) that acts on silencing of a target gene in an RNAi mechanism. Therefore, the CMTR1 of the present disclosure can be usefully used in the development of pharmaceuticals using siRNA as a therapeutic agent.

Cationic lipids comprising a steroidal moiety

Disclosed are cationic lipids which are compounds of Formula I. Cationic lipids provided herein can be useful for delivery and expression of mRNA and encoded protein, e.g., as a component of liposomal delivery vehicle, and accordingly can be useful for treating various diseases, disorders and conditions, such as those associated with deficiency of one or more proteins. ##STR00001##

Cationic lipids comprising a steroidal moiety

Disclosed are cationic lipids which are compounds of Formula I. Cationic lipids provided herein can be useful for delivery and expression of mRNA and encoded protein, e.g., as a component of liposomal delivery vehicle, and accordingly can be useful for treating various diseases, disorders and conditions, such as those associated with deficiency of one or more proteins. ##STR00001##

MICRORNA-MEDIATED METHODS FOR REJUVENATING CNS GLIAL POPULATIONS
20230212568 · 2023-07-06 ·

The present disclosure is directed to methods of inducing rejuvenation in a population of adult glial progenitor cells, and methods of treating a subject having a myelin deficiency. The method of inducing rejuvenation in a population of adult glial progenitor cells, may comprise: administering, to the population of adult glial progenitor cells, one or more nucleic acid molecules encoding microRNAs, wherein administering suppresses the signal transducer and activator of transcription 3 (STAT3) signaling pathway; and/or administering microRNAs, wherein administering suppresses the E2F transcription factor 6 (E2F6) signaling pathway; and/or administering microRNAs, wherein administering suppresses the Myc-associated factor X (MAX) signaling pathway, wherein said one or more nucleic acid molecules are administered in an amount sufficient to induce rejuvenation in the population of adult glial progenitor cells.

Methods for inducing cell division of postmitotic cells

The present disclosure provides methods for inducing cell cycle reentry of postmitotic cell. The present disclosure further provides cells and compositions for treating diseases, such as cardiovascular diseases, neural disorders, hearing loss, and diabetes.

Methods for inducing cell division of postmitotic cells

The present disclosure provides methods for inducing cell cycle reentry of postmitotic cell. The present disclosure further provides cells and compositions for treating diseases, such as cardiovascular diseases, neural disorders, hearing loss, and diabetes.

Methods for inducing cell division of postmitotic cells

The present disclosure provides methods for inducing cell cycle reentry of postmitotic cell. The present disclosure further provides cells and compositions for treating diseases, such as cardiovascular diseases, neural disorders, hearing loss, and diabetes.

Elimination of proliferating cells from stem cell-derived grafts

Provided herein are methods and compositions for a suicide gene approach comprising an expression vector comprising a cell cycle-dependent promoter driving the expression of a suicide gene. Also provided herein are methods to render proliferative cells sensitive to a prodrug after transplantation but avoids expression of the suicide gene in post-mitotic cells, such as neurons.

METHODS FOR TREATING VIRAL INFECTIONS

Provided herein are methods for preventing or treating a human immunodeficiency virus (HIV) infection or a simian immunodeficiency virus (SIV) infection in a subject. The methods include administering to the subject (a) a reservoir-depleting agent that binds to a host protein on a reservoir cell, and (b) an antiviral vaccine.