Patent ID: 11932856
Assignee: THE REGENTS OF THE UNIVERSITY OF CALIFORNIA
Field: Pharmaceuticals (Chemistry)
Classification: CPC C  A | IPC A  C

Claim 0:
1. A pharmaceutical composition in unit dose form comprising:
(a) a DNA vector encoding a guide RNA, wherein the guide RNA:
(i) comprises an antisense region that hybridizes to a target RNA, and
(ii) has two recruiting domains or no recruiting domains, wherein the two recruiting domains are recognized by an adenosine deaminase acting on RNA (ADAR) enzyme, and

(b) a pharmaceutically acceptable excipient, diluent, or carrier,, wherein the pharmaceutical composition does not encode or contain an exogenously added ADAR enzyme, wherein an endogenous ADAR enzyme present in endogenous amounts in a cell, when contacted with the guide RNA and the target RNA, deaminates an adenosine in the target RNA, and wherein the guide RNA, by leveraging endogenous ADAR enzyme in endogenous amounts, results in reduced off-target editing relative to pharmaceutical compositions that encode or contain exogenously added ADAR enzyme.