Title:
siRNA AND USE THEREOF
Document Type and Number:
WIPO Patent Application WO/2022/054801
Kind Code:
A1
Abstract:
The present invention provides: an siRNA that selectively suppresses the expression of FUS P525L point mutation; a pharmaceutical composition and ALS treatment agent containing the siRNA; and a screening method for the ALS treatment agent.
Inventors:
MORITA NAOKI (JP)
YAMAMOTO YOSHINOBU (JP)
MIYAZAKI KAZUKI (JP)
OKADA SUMIRE (JP)
YAMAMOTO YOSHINOBU (JP)
MIYAZAKI KAZUKI (JP)
OKADA SUMIRE (JP)
Application Number:
PCT/JP2021/032871
Publication Date:
March 17, 2022
Filing Date:
September 07, 2021
Export Citation:
Assignee:
OHARA PHARMACEUTICAL CO LTD (JP)
International Classes:
C12N5/10; A61K31/713; C12N15/113; C12N15/63; C12Q1/6806
Foreign References:
| JP2011528903A | 2011-12-01 | |||
| JP2015506905A | 2015-03-05 |
Other References:
VINCENT PICHER-MARTEL, PAUL N. VALDMANIS, PETER V. GOULD, JEAN-PIERRE JULIEN, NICOLAS DUPRÉ: "From animal models to human disease: a genetic approach for personalized medicine in ALS", ACTA NEUROPATHOLOGICA COMMUNICATIONS, vol. 4, no. 1, 1 December 2016 (2016-12-01), XP055403585, DOI: 10.1186/s40478-016-0340-5
POKRISHEVSKY EDWARD, GRAD LESLIE I., CASHMAN NEIL R.: "TDP-43 or FUS-induced misfolded human wild-type SOD1 can propagate intercellularly in a prion-like fashion", SCIENTIFIC REPORTS, vol. 6, no. 1, 1 April 2016 (2016-04-01), XP055910830, DOI: 10.1038/srep22155
POKRISHEVSKY EDWARD, GRAD LESLIE I., CASHMAN NEIL R.: "TDP-43 or FUS-induced misfolded human wild-type SOD1 can propagate intercellularly in a prion-like fashion", SCIENTIFIC REPORTS, vol. 6, no. 1, 1 April 2016 (2016-04-01), XP055910830, DOI: 10.1038/srep22155
Attorney, Agent or Firm:
Sha Patent and Trademark Firm (JP)
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