C12N2840/102

Method for expressing protein gene in response to expression of miRNA
11111503 · 2021-09-07 · ·

An mRNA forcibly expresses a protein gene in response to a miRNA, and a method for forcibly expressing the same, are provided. An artificial mRNA comprising a sequence encoding a protein gene, a miRNA target sequence linked to the 3′-terminal side of a Poly A sequence, and a translational repression sequence linked to the 3′-terminal side of the miRNA target sequence; and a method for expressing a protein gene in response to the expression of a miRNA, comprising a step of introducing the artificial mRNA into a cell.

ISOLATION OF NOVEL AAV'S AND USES THEREOF

The invention in some aspects relates to isolated nucleic acids, compositions, and kits useful for identifying adeno-associated viruses in cells. In some aspects, the invention provides kits and methods for producing somatic transgenic animal models using recombinant AAV (rAAV) to an animal having at least one transgene that expresses a small interfering nucleic acid or at least one binding site for a miRNA.

TREATING CANCER WITH VIRAL NUCLEIC ACID

This document provides methods and materials related to the use of nucleic acid coding for viruses to reduce the number of viable cancer cells within a mammal. For example, methods for using infectious nucleic acid to treat cancer, engineered viral nucleic acid, methods for making engineered viral nucleic acid, methods for identifying infectious nucleic acid for treating cancer, methods and materials for controlling virus-mediated cell lysis, and methods and materials for assessing the control of virus-mediated cell lysis are provided.

MESSENGER RNA COMPRISING FUNCTIONAL RNA ELEMENTS

The present disclosure provides messenger RNAs (mRNAs) having chemical and/or structural modifications, including RNA elements and/or modified nucleotides, in particular C-rich or CG-rich elements, which provide a desired translational regulatory activity to the mRNA.

MITOCHONDRIAL RNA IMPORT FOR TREATING MITOCHONDRIAL DISEASE

Disclosed is an RNA allotopic strategy to complement and genetically rescue mitochondrial gene defects. This approach can permit rescue of a mitochondrial DNA mutant by allotopic expression of a full-length recoded mitochondrial RNA that is transcribed in the nucleus, successfully imported into the mitochondria, and expressed there.

MICRORNA REGULATED EXPRESSION VECTORS, METHODS OF MAKING, AND USES THEREOF

Provided herein are vectors, compositions and methods for treating and diagnosing breast cancer. In exemplary embodiments, the present disclosure provides a vector for the expression of a therapeutic protein, wherein the vector comprises a microRNA binding domain (MBD) that facilitates the expression of the therapeutic protein in breast cancer cells and inhibits the expression of the therapeutic protein in non-breast cancer cells. The present disclosure also provides compositions comprising the vectors and methods of using the vectors for treating and/or diagnosing breast cancer.

Isolation of novel AAV's and uses thereof

The invention in some aspects relates to isolated nucleic acids, compositions, and kits useful for identifying adeno-associated viruses in cells. In some aspects, the invention provides kits and methods for producing somatic transgenic animal models using recombinant AAV (rAAV) to an animal having at least one transgene that expresses a small interfering nucleic acid or at least one binding site for a miRNA.

CONDITIONAL PROTEIN TRANSLATION SWITCHES, CONDITIONAL GENE EXPRESSION SYSTEMS AND USES THEREOF

Disclosed herein are protein translation switches and conditional gene expression systems that are compatible with retroviral and lentiviral gene delivery. The linking of a protein translation switch to a 3 gene of interest suppresses translation of the gene of interest, and the alteration of the protein translation switch by DNA recombinase-mediated DNA recombination relieves the suppressed translation of the 3 gene of interest. Also disclosed herein are methods of mimicking clinical pharmacology in a pre-clinical setting.

Viral Vector Production System
20200102578 · 2020-04-02 ·

The present invention relates to a nucleic acid sequence comprising a binding site operably linked to a nucleotide of interest, wherein the binding site is capable of interacting with an RNA-binding protein such that translation of the nucleotide of interest is repressed in a viral vector production cell.

Viral vector production system

The present invention relates to a nucleic acid sequence comprising a binding site operably linked to a nucleotide of interest, wherein the binding site is capable of interacting with an RNA-binding protein such that translation of the nucleotide of interest is repressed in a viral vector production cell.