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Last Updated: January 20, 2020

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Claims for Patent: 9,193,753

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Summary for Patent: 9,193,753
Title:RNA sequence-specific mediators of RNA interference
Abstract: The present invention relates to a Drosophila in vitro system which was used to demonstrate that dsRNA is processed to RNA segments 21-23 nucleotides (nt) in length. Furthermore, when these 21-23 nt fragments are purified and added back to Drosophila extracts, they mediate RNA interference in the absence of long dsRNA. Thus, these 21-23 nt fragments are the sequence-specific mediators of RNA degradation. A molecular signal, which may be their specific length, must be present in these 21-23 nt fragments to recruit cellular factors involved in RNAi. This present invention encompasses these 21-23 nt fragments and their use for specifically inactivating gene function. The use of these fragments (or chemically synthesized oligonucleotides of the same or similar nature) enables the targeting of specific mRNAs for degradation in mammalian cells, where the use of long dsRNAs to elicit RNAi is usually not practical, presumably because of the deleterious effects of the interferon response. This specific targeting of a particular gene function is useful in functional genomic and therapeutic applications.
Inventor(s): Tuschl; Thomas (Brooklyn, NY), Zamore; Phillip D. (Northborough, MA), Sharp; Phillip A. (Newton, MA), Bartel; David P. (Brookline, MA)
Assignee: University of Massachusetts (Boston, MA) Whitehead Institute for Biomedical Research (Cambridge, MA) Massachusetts Institute of Technology (Cambridge, MA) Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. (Munich, DE)
Application Number:13/830,751
Patent Claims: 1. A method of mediating RNA interference of an mRNA in a cell or organism, comprising: (a) introducing a double-stranded RNA molecule of from about 21 to 23 nucleotides which targets the mRNA for degradation into the cell or organism; (b) maintaining the cell or organism produced in (a) under conditions under which degradation of the mRNA occurs, thereby mediating RNA interference of the mRNA in the cell or organism.

2. The method of claim 1, wherein the RNA molecule of (a) is a chemically synthesized RNA or an analog of naturally occurring RNA.

3. The method of claim 1, wherein the mRNA is a cellular mRNA or a viral mRNA.

4. A method of mediating RNA interference of an mRNA in a cell or organism in which RNA interference occurs, comprising: (a) introducing into the cell or organism an RNA molecule of from about 21 to 23 nucleotides that mediates RNA interference of mRNA, wherein: (i) the RNA molecule comprises a sense strand and an antisense strand, and (ii) the antisense strand has sufficient sequence complementarity to the mRNA to mediate RNA interference of the mRNA; and (b) maintaining the cell or organism that contains the RNA molecule under conditions under which RNA interference occurs, thereby mediating RNA interference of the mRNA in the cell or organism.

5. The method of claim 4, wherein the RNA molecule is chemically synthesized RNA or an analog of RNA that mediates RNA interference.

6. The method of claim 4, wherein the mRNA is a cellular mRNA or a viral mRNA.

7. The method of claim 1, wherein the RNA molecule comprises a strand that has sufficient sequence correspondence to the mRNA to direct cleavage of the mRNA to which the sequence corresponds.

8. The method of claim 7, wherein the sufficient sequence correspondence to the mRNA is determined using a Drosophila lysate in vitro assay.

9. The method of claim 7, wherein the sufficient sequence correspondence to the mRNA is determined using a translation-based RNAi assay.

10. The method of claim 1, wherein a strand of the RNA molecule is about 21 nucleotides in length.

11. The method of claim 1, wherein a strand of the RNA molecule is from 21 nucleotides to 23 nucleotides in length.

12. The method of claim 1, wherein each strand of the RNA molecule is about 21 nucleotides in length.

13. The method of claim 1, wherein each strand of the RNA molecule is from 21 nucleotides to 23 nucleotides in length.

14. The method of claim 1, wherein the RNA molecule comprises one or more non-naturally occurring nucleotides.

15. The method of claim 1, wherein the RNA molecule comprises one or more deoxyribonucleotides.

16. The method of claim 14, wherein the RNA molecule comprises one or more non-standard nucleotides.

17. The method of claim 2, wherein the RNA molecule is a chemically synthesized RNA molecule.

18. The method of claim 2, wherein the analog of the RNA molecule differs from a naturally occurring RNA by the addition, deletion, substitution or alteration of one or more nucleotides.

19. The method of claim 18, wherein the alteration comprises addition of a non-nucleotide material to one or both ends of the RNA molecule.

20. The method of claim 1, wherein the mRNA is a cellular mRNA.

21. The method of claim 1, wherein the mRNA is a mammalian mRNA.

22. The method of claim 1, wherein the mRNA is a human mRNA.

23. The method of claim 1, wherein the mRNA encodes a protein whose presence is associated with a disease or an undesirable condition.

24. The method of claim 1, wherein the mRNA is a viral mRNA.

25. The method of claim 1, wherein the mRNA encodes an oncoprotein.

26. The method of claim 1, wherein the organism is a mammal.

27. The method of claim 4, wherein the sufficient sequence complementarity of the antisense strand to the mRNA is determined using a Drosophila lysate in vitro assay.

28. The method of claim 4, wherein the sufficient sequence complementarity of the antisense strand to the mRNA is determined using a translation-based RNAi assay.

29. The method of claim 4, wherein the RNA molecule is a chemically synthesized RNA.

30. The method of claim 4, wherein the RNA molecule is an analog of naturally occurring RNA.

31. The method of claim 30, wherein the analog of the RNA molecule differs from a naturally occurring RNA by the addition, deletion, substitution or alteration of one or more nucleotides.

32. The method of claim 31, wherein the alteration comprises addition of a non-nucleotide material to one or both ends of the RNA molecule.

33. The method of claim 4, wherein a strand of the RNA molecule is from 21 nucleotides to 23 nucleotides in length.

34. The method of claim 4, wherein one or both strands of the RNA molecule is about 21 nucleotides in length.

35. The method of claim 4, wherein each strand of the RNA molecule is from 21 nucleotides to 23 nucleotides in length.

36. The method of claim 4, wherein the RNA molecule comprises one or more non-naturally occurring nucleotides.

37. The method of claim 4, wherein the RNA molecule comprises one or more deoxyribonucleotides.

38. The method of claim 4, wherein the RNA molecule comprises one or more non-standard nucleotides.

39. The method of claim 4, wherein the mRNA is a cellular mRNA.

40. The method of claim 4, wherein the mRNA is a mammalian mRNA.

41. The method of claim 4, wherein the mRNA is a human mRNA.

42. The method of claim 4, wherein the mRNA encodes a protein whose presence is associated with a disease or an undesirable condition.

43. The method of claim 4, wherein the mRNA is a viral mRNA.

44. The method of claim 4, wherein the mRNA encodes an oncoprotein.

45. The method of claim 4, wherein the organism is a mammal.

46. The method of claim 1, wherein the double-stranded RNA molecule comprises two separate strands which are not covalently linked.

47. The method of claim 4, wherein the double-stranded RNA molecule comprises two separate strands which are not covalently linked.

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