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US 12,275,941 B2Granted
Products and methods for inhibition of expression of dynamin-1 variants
AssigneesResearch Institute At Nationwide Children's Hospital, The Trustees of Columbia University in the City of New York
InventorsScott Quenton Harper, Wayne N. Frankel
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Abstract
RNA interference-based methods and products for inhibiting the expression of pathogenic dynamin-1 variants are provided. Delivery vehicles such as recombinant adeno-associated viruses deliver DNAs encoding RNAs that inhibit the expression of the dynamin-1 variants. The methods treat, for example, developmental and epileptic encephalopathies.
Claims25
A nucleic acid comprising
- (a) a polynucleotide sequence comprising at least 82%, 83%, 84%, 85%, 86%, 87%, 88%, 89%, 90%, 91%, 92%, 93%, 94%, 95%, 96%, 97%, 98%, 99%, or 100% identity to the dynamin-1 (DNM1) artificial inhibitory RNA-encoding polynucleotide sequence set forth in any one of SEQ ID NOs:
- 1-17;
- (b) a polynucleotide sequence encoding a DNM1 artificial inhibitory RNA comprising the artificial inhibitory RNA polynucleotide sequence set forth in any one of SEQ ID NOs:
- 18-34;
- or
- (c) a polynucleotide sequence encoding a mature DNM1 antisense guide strand comprising the polynucleotide sequence set forth in any one of SEQ ID NO:
- 35-51.
4 more elements
Dependencies are inferred from the claim text.