The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.
The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.
Patent No.:
Date of Patent:
Oct. 10, 2023
Filed:
Jul. 28, 2020
Genzyme Corporation, Cambridge, MA (US);
Lisa M. Stanek, Natick, MA (US);
Adam Palermo, Boston, MA (US);
Brenda Richards, Hopkinton, MA (US);
Sergio Pablo Sardi, Newton, MA (US);
Catherine O′Riordan, Bridgewater, NJ (US);
Antonius Song, Dublin, CA (US);
Genzyme Corporation, Cambridge, MA (US);
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.