Patent ID: 11959082
Assignee: NORTHWESTERN UNIVERSITY
Field: Biotechnology (Chemistry)
Classification: CPC C  A | IPC A  C

Claim 0:
1. An shRNA polynucleotide comprising a dsRNA sequence having a first strand, otherwise referred to as an “A” strand, and a second strand, otherwise referred to as a “B” strand, the dsRNA defined as follows:, 5′-A01 A02 A03 A04 A05 A06 A07 A08 A09 A10 A11 A12 

    |   |   |   |   |   |   |   |   |   |   |   |   

3′-B21 B20 B19 B18 B17 B16 B15 B14 B13 B12 B11 B10 

 

            A13 A14 A15 A16 A17 A18 A19 A20 A21-3′

             |   |   |   |   |   |   |   |   |

            B09 B08 B07 B06 B05 B04 B03 B02 B01-5′

wherein:
A01 through A21 and B01 through B21 are any ribonucleotide selected from A, U, G, and C, provided that:
(i) A01-A21 are complementary to B01-B21, respectively;
(ii) A01 and B01 are A or U;
(iii) one of the A strand from A02-A07 and the B strand from B02-B07 comprises the sequence GGGGGC; and
(iv) the other of the A strand and the B strand comprises a trinucleotide repeat sequence (CAG)n, wherein n is an integer from 3-10;
wherein the 3′ end of the A strand is linked via polynucleotides to the 5′ end of the B strand; and
wherein both of the A strand and the B strand are toxic after the shRNA is introduced into a cell and the shRNA is processed for RNA interference, wherein the dsRNA sequence comprises:, (SEQ ID NOs: 47 and 48)

5′-CAGCAGCAGCAGCCCCC-3′

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3′-GUCGUCGUCGUCGGGGG-5′.