Title:
血液脳関門通過型ヘテロ２本鎖核酸
Document Type and Number:
Japanese Patent JP7043078
Kind Code:
B2
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.
Inventors:
Takanori Yokota
Tetsuya Nagata
Tetsuya Nagata
Application Number:
JP2018540337A
Publication Date:
March 29, 2022
Filing Date:
September 25, 2017
Export Citation:
Assignee:
National University Corporation Tokyo Medical and Dental University
International Classes:
C12N15/113; A61K9/08; A61K31/7088; A61K31/711; A61K31/713; A61K47/54; A61K48/00; A61P25/00; A61P27/02
Domestic Patent References:
| JP2015502134A |
Foreign References:
| WO2015075942A1 |
Other References:
UNO,Y. et al.,Efficient In Vivo delivery of siRNA to the Brain by Conjugation of alpha-Tocopherol.,第32 回日本分子生物学会年 会プログラム・講演要旨集,2009年,p.248,1P-0816
UNO,Y. et al.,Efficient In Vivo Delivery of siRNA to the Brain by Conjugation of Alpha-Tocopherol.,Molecular Therapy,2009年,Vol.17, Supplement 1,p.S197, 515
NISHINA,K. et al.,Novel oligonucleotide based on DNA/RNA heteroduplex structures.,第57 回日本神経学会学術大会 プログ ラム・抄録集,2016年,p.194
UNO,Y. et al.,Efficient In Vivo Delivery of siRNA to the Brain by Conjugation of Alpha-Tocopherol.,Molecular Therapy,2009年,Vol.17, Supplement 1,p.S197, 515
NISHINA,K. et al.,Novel oligonucleotide based on DNA/RNA heteroduplex structures.,第57 回日本神経学会学術大会 プログ ラム・抄録集,2016年,p.194
Attorney, Agent or Firm:
Hiraki International Patent Office