C12Y301/26003

OPTIDICER CONSTRUCT FOR AGE-RELATED MACULAR DEGENERATION

Provided are nucleotide sequences encoding polypeptides with ribonuclease III activity, wherein the nucleotide sequences have been modified to reduce their regulation by miRNAs. In some embodiments, the nucleotide sequences are at least 50% and as much as 100% identical to SEQ ID NO: 20 or SEQ ID NO: 22, and/or encode polypeptides that are at least 90% percent identical to SEQ ID NO: 23. Also provided are vectors and host cells that include the nucleotide sequences, methods for expressing the nucleotide sequences in cells, tissues, and organs, which in some embodiments can be in the eye of a subject in need thereof, methods for preventing and/or treating development of diseases or disorders and/or for restoring undesirably low DICER1 expression using the nucleotide sequences, and pharmaceutical compositions that have the presently disclosed nucleotide sequences.

SYSTEMS, METHODS AND COMPOSITION OF USING RNASE III MUTANTS TO PRODUCE SRNA TO CONTROL HOST PATHOGEN INFECTION
20230354822 · 2023-11-09 ·

The current invention includes systems, methods and compositions for the generation of sRNA molecules using select RNase III mutants. In one preferred embodiment, invention includes systems, methods and compositions for the generation of sRNA molecules using RNase III mutants to control a host pathogen through the production and diffusion of sRNA molecules that may initiate an RNAi pathway response directed to a host pathogen. Additional embodiments of the current invention include systems, methods and compositions for the DICER-independent generation of sRNA molecules using select RNase III mutants.

Systems, methods and composition of using RNase III mutants to produce sRNA to control host pathogen infection

The current invention includes systems, methods and compositions for the generation of sRNA molecules using select RNase III mutants. In one preferred embodiment, invention includes systems, methods and compositions for the generation of sRNA molecules using RNase III mutants to control a host pathogen through the production and diffusion of sRNA molecules that may initiate an RNAi pathway response directed to a host pathogen. Additional embodiments of the current invention include systems, methods and compositions for the DICER-independent generation of sRNA molecules using select RNase III mutants.

MAKING AND USING IN VITRO-SYNTHESIZED ssRNA FOR INTRODUCING INTO MAMMALIAN CELLS TO INDUCE A BIOLOGICAL OR BIOCHEMICAL EFFECT

The present invention relates to compositions, kits and methods for making and using RNA compositions comprising in vitro-synthesized ssRNA inducing a biological or biochemical effect in a mammalian cell or organism into which the RNA composition is repeatedly or continuously introduced. In certain embodiments, the invention provides compositions and methods for changing the state of differentiation or phenotype of a human or other vertebrate cell. For example, the present invention provides mRNA and methods for reprogramming cells that exhibit a first differentiated state or phenotype to cells that exhibit a second differentiated state or phenotype, such as to reprogram human somatic cells to pluripotent stem cells.

RNA AFFINITY PURIFICATION
20220290125 · 2022-09-15 · ·

Provided herein, in some embodiments, are methods of purifying a nucleic acid preparation. The methods may comprise contacting a nucleic acid preparation comprising messenger ribonucleic acid with an RNase III enzyme that is immobilized on a solid support and binds to double-stranded RNA contaminants.

RNA affinity purification
11384352 · 2022-07-12 · ·

Provided herein, in some embodiments, are methods of purifying a nucleic acid preparation. The methods may comprise contacting a nucleic acid preparation comprising messenger ribonucleic acid with an RNase III enzyme that is immobilized on a solid support and binds to double-stranded RNA contaminants.

Making and using in vitro-synthesized ssRNA for introducing into mammalian cells to induce a biological or biochemical effect

The present invention relates to compositions, kits and methods for making and using RNA compositions comprising in vitro-synthesized ssRNA inducing a biological or biochemical effect in a mammalian cell or organism into which the RNA composition is repeatedly or continuously introduced. In certain embodiments, the invention provides compositions and methods for changing the state of differentiation or phenotype of a human or other vertebrate cell. For example, the present invention provides mRNA and methods for reprogramming cells that exhibit a first differentiated state or phenotype to cells that exhibit a second differentiated state or phenotype, such as to reprogram human somatic cells to pluripotent stem cells.

METHODS AND COMPOSITIONS FOR TREATING CANCER
20210299234 · 2021-09-30 ·

Embodiments are directed to: (i) neutrophil secreted factors that have the capacity to kill a broad range of cancer cells without affecting the viability of non-cancer cells. Two neutrophil killing factors have been identified by the inventors: (1) eosinophil cationic protein (ECP) and (2) neutrophil elastase (ELANE); or (ii) therapeutic compositions that include CD95 degrading polypeptide components and methods of treating cancer with the same.

Systems, Methods And Composition of Using RNASE III Mutants to Produce sRNA to Control Host Pathogen Infection
20210017523 · 2021-01-21 ·

The current invention includes systems, methods and compositions for the generation of sRNA molecules using select RNase III mutants. In one preferred embodiment, invention includes systems, methods and compositions for the generation of sRNA molecules using RNase III mutants to control a host pathogen through the production and diffusion of sRNA molecules that may initiate an RNAi pathway response directed to a host pathogen. Additional embodiments of the current invention include systems, methods and compositions for the DICER-independent generation of sRNA molecules using select RNase III mutants.

MEDIATORS OF GENE SILENCING
20240011037 · 2024-01-11 ·

The present invention relates to a method of inhibiting expression of a gene in a biological system. The method of the present invention comprises introducing a tRNA-derived polynucleotide into the biological system. The tRNA-derived polynucleotide of 5 the present invention comprises a sequence that is complementary to an intronic region or exonic region of the gene whose expression is to be inhibited.