Invention Grant
- Patent Title: Blood-brain barrier permeable heteroduplex nucleic acid
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Application No.: US16335017Application Date: 2017-09-25
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Publication No.: US11433089B2Publication Date: 2022-09-06
- Inventor: Takanori Yokota , Tetsuya Nagata
- Applicant: NATIONAL UNIVERSITY CORPORATION TOKYO MEDICAL AND DENTAL UNIVERSITY
- Applicant Address: JP Tokyo
- Assignee: NATIONAL UNIVERSITY CORPORATION TOKYO MEDICAL AND DENTAL UNIVERSITY
- Current Assignee: NATIONAL UNIVERSITY CORPORATION TOKYO MEDICAL AND DENTAL UNIVERSITY
- Current Assignee Address: JP Tokyo
- Agency: Lucas & Mercanti, LLP
- Priority: JPJP2016-185806 20160923
- International Application: PCT/JP2017/034561 WO 20170925
- International Announcement: WO2018/056442 WO 20180329
- Main IPC: A61K48/00
- IPC: A61K48/00 ; A61K31/711 ; A61K47/22 ; A61K47/28 ; C12N15/113 ; A61K47/54 ; A61P25/00 ; A61K31/7088 ; C12N15/09 ; A61P27/02 ; A61K9/00 ; A61K31/7125

Abstract:
The present invention provides a composition and a method for efficiently delivering a nucleic acid agent to the central nervous system and/or the retina and bringing about antisense effects. Provided is a composition for reducing the expression level of a target transcription product in the central nervous system and/or retina of a subject, the composition including a nucleic acid complex that includes a first nucleic acid strand and a second nucleic acid strand, wherein: the first nucleic acid strand comprises a base sequence capable of hybridizing with at least part of the target transcription product and has an antisense effect on the target transcription product; the second nucleic acid strand comprises a base sequence complementary to the first nucleic acid strand and is conjugated to tocopherol, cholesterol, or an analog thereof; and the first nucleic acid strand is annealed to the second nucleic acid strand.
Public/Granted literature
- US20190247414A1 BLOOD-BRAIN BARRIER PERMEABLE HETERODUPLEX NUCLEIC ACID Public/Granted day:2019-08-15
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