TW202221121A - 抑制plp1表現之組成物及方法 - Google Patents

抑制plp1表現之組成物及方法 Download PDF

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TW202221121A
TW202221121A TW110128729A TW110128729A TW202221121A TW 202221121 A TW202221121 A TW 202221121A TW 110128729 A TW110128729 A TW 110128729A TW 110128729 A TW110128729 A TW 110128729A TW 202221121 A TW202221121 A TW 202221121A
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seq
nos
nucleotides
plp1
oligonucleotide
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TW110128729A
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Chinese (zh)
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鮑伯 布朗
邁爾 奧斯本
阿爾門 茜吉蘭
張春陽
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美商黛瑟納製藥公司
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TW110128729A 2020-08-04 2021-08-04 抑制plp1表現之組成物及方法 TW202221121A (zh)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US202063061040P 2020-08-04 2020-08-04
US63/061,040 2020-08-04
US202163151445P 2021-02-19 2021-02-19
US63/151,445 2021-02-19

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TW202221121A true TW202221121A (zh) 2022-06-01

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US (1) US20240084309A1 (https=)
EP (1) EP4192478A2 (https=)
JP (1) JP2023538284A (https=)
KR (1) KR20230058412A (https=)
AU (1) AU2021321430A1 (https=)
CA (1) CA3190481A1 (https=)
CL (1) CL2023000371A1 (https=)
IL (1) IL300286A (https=)
MX (1) MX2023001450A (https=)
TW (1) TW202221121A (https=)
WO (1) WO2022031847A2 (https=)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4172338A4 (en) 2020-06-29 2025-06-11 Ionis Pharmaceuticals, Inc. COMPOUNDS AND METHODS FOR MODULATING PLP1
EP4499831A2 (en) * 2022-03-24 2025-02-05 Ionis Pharmaceuticals, Inc. Rnai agents of modulating plp1
CN119173631A (zh) * 2022-05-12 2024-12-20 迪克纳制药公司 用于抑制mapt表达的组合物和方法
IL316843A (en) * 2022-05-13 2025-01-01 Dicerna Pharmaceuticals Inc Compounds and methods for inhibiting SNCA deactivation
WO2024040041A1 (en) * 2022-08-15 2024-02-22 Dicerna Pharmaceuticals, Inc. Regulation of activity of rnai molecules
TW202430192A (zh) * 2022-10-11 2024-08-01 德商百靈佳殷格翰國際股份有限公司 用於治療nash之劑量方案
TW202430191A (zh) * 2022-10-11 2024-08-01 德商百靈佳殷格翰國際股份有限公司 用於治療伴有晚期纖維化及/或硬化之nash的方法
WO2024097821A1 (en) * 2022-11-02 2024-05-10 Sanegene Bio Usa Inc. Nucleotide-based enhancement agent for rna delivery and therapy

Family Cites Families (26)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6469158B1 (en) 1992-05-14 2002-10-22 Ribozyme Pharmaceuticals, Incorporated Synthesis, deprotection, analysis and purification of RNA and ribozymes
US5977343A (en) 1992-05-14 1999-11-02 Ribozyme Pharmaceuticals, Inc. Synthesis, deprotection, analysis and purification of RNA and ribozymes
US5804683A (en) 1992-05-14 1998-09-08 Ribozyme Pharmaceuticals, Inc. Deprotection of RNA with alkylamine
ATE227342T1 (de) 1993-09-02 2002-11-15 Ribozyme Pharm Inc Enzymatische nukleiksaüre die nicht-nukleotide enthaltet
US5889136A (en) 1995-06-09 1999-03-30 The Regents Of The University Of Colorado Orthoester protecting groups in RNA synthesis
US5998203A (en) 1996-04-16 1999-12-07 Ribozyme Pharmaceuticals, Inc. Enzymatic nucleic acids containing 5'-and/or 3'-cap structures
US6111086A (en) 1998-02-27 2000-08-29 Scaringe; Stephen A. Orthoester protecting groups
CA2429814C (en) 2000-12-01 2014-02-18 Thomas Tuschl Rna interference mediating small rna molecules
US20050159378A1 (en) 2001-05-18 2005-07-21 Sirna Therapeutics, Inc. RNA interference mediated inhibition of Myc and/or Myb gene expression using short interfering nucleic acid (siNA)
WO2003040395A2 (en) 2001-11-07 2003-05-15 Applera Corporation Universal nucleotides for nucleic acid analysis
AU2003295600A1 (en) * 2002-11-14 2004-06-15 Dharmacon, Inc. Functional and hyperfunctional sirna
US20070265220A1 (en) 2004-03-15 2007-11-15 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
WO2005116204A1 (ja) * 2004-05-11 2005-12-08 Rnai Co., Ltd. Rna干渉を生じさせるポリヌクレオチド、および、これを用いた遺伝子発現抑制方法
WO2008020435A2 (en) * 2006-08-15 2008-02-21 Quark Pharmaceuticals, Inc Compositions and methods for treatment of mood disorders
WO2009099991A2 (en) * 2008-01-31 2009-08-13 The Brigham And Women's Hospital, Inc. Treatment of cancer
DK2341943T3 (en) * 2008-09-22 2019-02-25 Dicerna Pharmaceuticals Inc COMPOSITIONS AND METHODS OF SPECIFIC INHIBITION OF DSRNA REPRINT WITH MODIFICATIONS
US20100249214A1 (en) 2009-02-11 2010-09-30 Dicerna Pharmaceuticals Multiplex dicer substrate rna interference molecules having joining sequences
KR101728655B1 (ko) 2008-12-18 2017-04-19 다이서나 파마수이티컬, 인크. 유전자 발현의 특이적 억제를 위한 연장된 다이서 기질 제제 및 방법
WO2011005860A2 (en) 2009-07-07 2011-01-13 Alnylam Pharmaceuticals, Inc. 5' phosphate mimics
WO2011133871A2 (en) 2010-04-22 2011-10-27 Alnylam Pharmaceuticals, Inc. 5'-end derivatives
AU2012328680A1 (en) 2011-10-25 2014-05-01 Ionis Pharmaceuticals, Inc. Antisense modulation of GCCR expression
DK3569711T3 (da) 2014-12-15 2021-02-22 Dicerna Pharmaceuticals Inc Ligandmodificerede dobbeltstrengede nukleinsyrer
HUE059718T2 (hu) 2016-09-02 2022-12-28 Dicerna Pharmaceuticals Inc 4'-foszfát analógok és azokat tartalmazó oligonukleotidok
EP3551754B1 (en) * 2016-12-08 2023-08-30 Case Western Reserve University Methods and compositions for enhancing functional myelin production
TW201945009A (zh) * 2018-02-07 2019-12-01 國立研究開發法人國立精神 神經醫療研究中心 寡樹突細胞專一性啟動子、對PLP1基因具專一性之miRNA、包含該啟動子及/或該miRNA之載體與包含該載體之醫藥組合物
WO2020123083A1 (en) * 2018-12-12 2020-06-18 Dicerna Pharmaceuticals, Inc. Double-stranded nucleic acid inhibitor molecules containing a triloop

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WO2022031847A3 (en) 2022-04-14
KR20230058412A (ko) 2023-05-03
CA3190481A1 (en) 2022-02-10
EP4192478A2 (en) 2023-06-14
IL300286A (en) 2023-04-01
MX2023001450A (es) 2023-04-14
AU2021321430A1 (en) 2023-03-02
JP2023538284A (ja) 2023-09-07
US20240084309A1 (en) 2024-03-14
CL2023000371A1 (es) 2023-10-13

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