RNAi induced huntingtin gene suppression
A technology of gene therapy and DNA sequence, applied in DNA/RNA fragment, gene therapy, genetic engineering, etc., can solve the problem of knocking down huntingtin gene
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[0061] 1. A double-stranded RNA comprising a first RNA sequence and a second RNA sequence, wherein the first RNA sequence and the second RNA sequence are substantially complementary, wherein the first RNA sequence has at least 19 nucleotides The sequence length is basically complementary to SEQID NO.1.
[0062] 2. The double-stranded RNA according to embodiment 1, wherein the double-stranded RNA is capable of reducing the expression of the huntingtin gene.
[0063] 3. The double-stranded RNA according to embodiment 1 or 2, wherein said double-stranded RNA is comprised within a pre-miRNA scaffold, pri-miRNA scaffold, shRNA or siRNA, preferably within a pre-miRNA scaffold.
[0064]4. The double-stranded RNA according to any one of embodiments 1-3, wherein said first RNA sequence has a sequence length of at least 20 nucleotides, preferably at least 21 nucleotides.
[0065] 5. The double-stranded RNA according to any one of embodiments 1-4, wherein the first RNA sequence is fully...
Embodiment
[0078] miRNA scaffold expression constructs & siRNA
[0079] In order to construct the miRNA scaffold vector based on miR155, it will be combined with figure 1 The selected target sequence in the indicated HTT is fully complementary with a 21 nucleotide (bp) sequence embedded into the pri-mir of pcDNA6.2-GW / EmGFP-miR (Invitrogen, Carlsbad, CA) pVD-CMV-miHTT-155 ( Figure 2C Examples of expression cassette sequences are shown, and the pre-miRNA and pri-miRNA sequences contained in the expressed RNA are shown in Figure 2B and Figure 2F middle). Based on the instructions provided by Invitrogen (BLOCK-iT, Pol II miR RNAi expression vector kit, Verson E, June 22, 2007, 25-0857), through the BsaI site pairing in pcDNA6.2-GW / emGFP-mir155 Synthetic double-stranded oligonucleotides were annealed to design the pri-mir-155 construct. Using the Mfold software (Nucleic Acids Res. 31(13), 3406-15, (2003)), it was verified that Structures of all artificial pre-miRNAs encoded by miHtt...
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