City of Hope

Mina Therapeutics Limited

Pal Saetrom

John J Rossi

Ali Ehsani

Jane Zhang

Lars Aagaard

Ola Snove

Britta Vallazza

Britta Hoehn

The present invention relates to novel short interfering RNA (siRNA) molecules that are multi-targeted. More specifically, the present invention relates to siRNA molecules that target two or more sequences. In one embodiment, multi-targeting siRNA molecules are designed to incorporate features of siRNA molecules and features of micro-RNA (miRNA) molecules. In another embodiment, multi-targeting siRNA molecules are designed so that each strand is directed to separate targets.

Multi-targeting short interfering RNAs

The present invention provides a method of inducing insulin production in a cell by up-regulating a target gene involved in insulin production in said cell using an saRNA (short activating ribonucleic acid) which specifically down-regulates a target antisense RNA transcript present in said cell, wherein (i) said target RNA transcript is complementary to a sequence located on the coding strand of the target gene between 1000 nucleotides upstream and 1000 nucleotides downstream of the transcription start site of the target gene; and (ii) said sRNA is a single or double stranded RNA molecule up to 30 nucleotides in length comprising a sequence of at least 13 nucleotides which has at least 95% complementarity to a region of the target transcript. Also provided are certain saRNA molecules and uses thereof, particular medical uses, and induced cells and uses thereof.

Methods of inducing insulin production

The present invention provides a short RNA capable of upregulating MafA expression in a human cell, wherein the short RNA includes a first strand which is 19 to 25 nucleotides in length and comprises the sequence AUCUGUACUGGAUGAGCGG (SEQ ID NO:1) or UUUCCCGCAGGAGAUUGAC (SEQ ID NO:2). Also provided are uses of the short RNA, particular medical uses, and induced cells and uses of the induced cells.

RNA molecules that upregulate insulin production

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Trondheim, Norway

Pal Saetrom