Title:
BLOOD-BRAIN BARRIER PERMEABLE HETERODUPLEX NUCLEIC ACID
Document Type and Number:
WIPO Patent Application WO/2018/056442
Kind Code:
A1
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 includes 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 includes a base sequence complementary to the first nucleic acid strand and is bonded to tocopherol, cholesterol, or an analog thereof; and the first nucleic acid strand is annealed to the second nucleic acid strand.
Inventors:
YOKOTA TAKANORI (JP)
NAGATA TETSUYA (JP)
NAGATA TETSUYA (JP)
Application Number:
PCT/JP2017/034561
Publication Date:
March 29, 2018
Filing Date:
September 25, 2017
Export Citation:
Assignee:
UNIV NAT CORP TOKYO MEDICAL & DENTAL (JP)
International Classes:
C12N15/09; A61K31/7088; A61K31/711; A61K47/22; A61K47/28; A61K47/50; A61K48/00; A61P25/00; C12N15/113
Domestic Patent References:
| WO2015075942A1 | 2015-05-28 |
Foreign References:
| JP2015502134A | 2015-01-22 |
Other References:
UNO, Y. ET AL.: "Efficient In Vivo delivery of siRNA to the Brain by Conjugation of alpha-Tocopherol", MOLECULAR THERAPY,20090501BERLIN/HEIDELBERG, vol. 17, no. 1, 1 May 2009 (2009-05-01), pages S197, XP055483410
UNO, Y. ET AL.: "Efficient In Vivo Delivery of siRNA to the Brain by Conjugation of Alpha-Tocopherol", MOLECULAR THERAPY, vol. 17, no. 1, 2009, pages S197, XP055483410
NISHINA, K. ET AL.: "Novel oligonucleotide based on DNA/RNA heteroduplex structures", 57TH ANNUAL MEETING OF THE JAPANESE SOCIETY OF NEUROLOGY: ABSTRACTS, vol. 56, no. S65, May 2016 (2016-05-01), pages 194, XP009516447
UNO, Y. ET AL.: "Efficient In Vivo Delivery of siRNA to the Brain by Conjugation of Alpha-Tocopherol", MOLECULAR THERAPY, vol. 17, no. 1, 2009, pages S197, XP055483410
NISHINA, K. ET AL.: "Novel oligonucleotide based on DNA/RNA heteroduplex structures", 57TH ANNUAL MEETING OF THE JAPANESE SOCIETY OF NEUROLOGY: ABSTRACTS, vol. 56, no. S65, May 2016 (2016-05-01), pages 194, XP009516447
Attorney, Agent or Firm:
HIRAKI & ASSOCIATES (JP)
Download PDF:
Previous Patent: AXIS DEVIATION ESTIMATING DEVICE
Next Patent: CAPACITANCE DETECTION DEVICE AND IMAGE READING DEVICE
Next Patent: CAPACITANCE DETECTION DEVICE AND IMAGE READING DEVICE