Title:
VARIANT RNAi
Document Type and Number:
WIPO Patent Application WO/2016/130589
Kind Code:
A3
Abstract:
Provided herein are RNAi molecules including a first strand containing a guide sequence and a second strand comprising a non-guide sequence where the non-guide sequence contains a bulge opposite the seed region of the guide sequences; e.g., opposite the cleavage sequence. In some aspects, the invention provides RNAi for treating Huntington's disease. Further provided herein are expression cassettes, vectors (e.g., rAAV, recombinant adenoviral, recombinant lentiviral, and recombinant HSV vectors), cells, viral particles, and pharmaceutical compositions containing the RNAi. Yet further provided herein are methods and kits related to the use of the RNAi, for example, to treat Huntington' s disease.
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Inventors:
STANEK LISA M (US)
PALERMO ADAM (US)
RICHARDS BRENDA (US)
SARDI SERGIO PABLO (US)
O'RIORDAN CATHERINE (US)
SONG ANTONIUS (US)
PALERMO ADAM (US)
RICHARDS BRENDA (US)
SARDI SERGIO PABLO (US)
O'RIORDAN CATHERINE (US)
SONG ANTONIUS (US)
Application Number:
PCT/US2016/017207
Publication Date:
September 15, 2016
Filing Date:
February 09, 2016
Export Citation:
Assignee:
GENZYME CORP (US)
International Classes:
A61P25/28; C12N15/113
Domestic Patent References:
| WO2011034811A1 | 2011-03-24 | |||
| WO2009100502A1 | 2009-08-20 | |||
| WO2004029212A2 | 2004-04-08 |
Other References:
SHUO GU ET AL: "The Loop Position of shRNAs and Pre-miRNAs Is Critical for the Accuracy of Dicer Processing In Vivo", CELL, vol. 151, no. 4, 1 November 2012 (2012-11-01), US, pages 900 - 911, XP055268566, ISSN: 0092-8674, DOI: 10.1016/j.cell.2012.09.042
MIYAGISHI M ET AL: "Optimization of an siRNA-expression systems with an improved hairpin and its significant suppressive effects in mammalian cells", JOURNAL OF GENE MEDICINE, JOHN WILEY & SONS, INC, US, vol. 6, 1 January 2004 (2004-01-01), pages 715 - 723, XP003010166, ISSN: 1099-498X, DOI: 10.1002/JGM.556
P. R. CLARK ET AL: "Knockdown of TNFR1 by the sense strand of an ICAM-1 siRNA: dissection of an off-target effect", NUCLEIC ACIDS RESEARCH, vol. 36, no. 4, 1 January 2007 (2007-01-01), pages 1081 - 1097, XP055000172, ISSN: 0305-1048, DOI: 10.1093/nar/gkm630
GANTIER MICHAEL P ET AL: "Rational design of immunostimulatory siRNAs", MOLECULAR THERAPY, NATURE PUBLISHING GROUP, GB, vol. 18, no. 4, 1 April 2010 (2010-04-01), pages 785 - 795, XP008123336, ISSN: 1525-0024, [retrieved on 20100202], DOI: 10.1038/MT.2010.4
CHIU Y-L ET AL: "RNAi in Human Cells: Basic Structural and Functional Features of Small Interfering RNA", MOLECULAR CELL, CELL PRESS, CAMBRIDGE, MA, US, vol. 10, 1 September 2002 (2002-09-01), pages 549 - 561, XP002978510, ISSN: 1097-2765, DOI: 10.1016/S1097-2765(02)00652-4
MIYAGISHI M ET AL: "Optimization of an siRNA-expression systems with an improved hairpin and its significant suppressive effects in mammalian cells", JOURNAL OF GENE MEDICINE, JOHN WILEY & SONS, INC, US, vol. 6, 1 January 2004 (2004-01-01), pages 715 - 723, XP003010166, ISSN: 1099-498X, DOI: 10.1002/JGM.556
P. R. CLARK ET AL: "Knockdown of TNFR1 by the sense strand of an ICAM-1 siRNA: dissection of an off-target effect", NUCLEIC ACIDS RESEARCH, vol. 36, no. 4, 1 January 2007 (2007-01-01), pages 1081 - 1097, XP055000172, ISSN: 0305-1048, DOI: 10.1093/nar/gkm630
GANTIER MICHAEL P ET AL: "Rational design of immunostimulatory siRNAs", MOLECULAR THERAPY, NATURE PUBLISHING GROUP, GB, vol. 18, no. 4, 1 April 2010 (2010-04-01), pages 785 - 795, XP008123336, ISSN: 1525-0024, [retrieved on 20100202], DOI: 10.1038/MT.2010.4
CHIU Y-L ET AL: "RNAi in Human Cells: Basic Structural and Functional Features of Small Interfering RNA", MOLECULAR CELL, CELL PRESS, CAMBRIDGE, MA, US, vol. 10, 1 September 2002 (2002-09-01), pages 549 - 561, XP002978510, ISSN: 1097-2765, DOI: 10.1016/S1097-2765(02)00652-4
Attorney, Agent or Firm:
DONAHUE, Brian, A. et al. (755 Page Mill RoadPalo Alto, CA, US)
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