CN105247052A - B-细胞cll/淋巴瘤11a(bcl11a)的寡核苷酸调节剂及其用途 - Google Patents

B-细胞cll/淋巴瘤11a(bcl11a)的寡核苷酸调节剂及其用途 Download PDF

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Publication number
CN105247052A
CN105247052A CN201480029327.XA CN201480029327A CN105247052A CN 105247052 A CN105247052 A CN 105247052A CN 201480029327 A CN201480029327 A CN 201480029327A CN 105247052 A CN105247052 A CN 105247052A
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seqidno
bcl11a
lna
antisense oligonucleotide
nucleotide
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马伊·黑特耶恩
尼尔斯·菲斯克尔·尼尔森
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Copenhagen Co Ltd Of Roche Innovation Center
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Copenhagen Co Ltd Of Roche Innovation Center
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    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P7/06—Antianaemics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
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    • C12N2310/10—Type of nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/31—Chemical structure of the backbone
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
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    • C12N2310/30—Chemical structure
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CN201480029327.XA 2013-05-24 2014-05-26 B-细胞cll/淋巴瘤11a(bcl11a)的寡核苷酸调节剂及其用途 Pending CN105247052A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361827484P 2013-05-24 2013-05-24
US61/827,484 2013-05-24
PCT/EP2014/060813 WO2014188001A1 (en) 2013-05-24 2014-05-26 Oligonucleotide modulators of b-cell cll/lymphoma 11a (bcl11a) and uses thereof

Publications (1)

Publication Number Publication Date
CN105247052A true CN105247052A (zh) 2016-01-13

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CN201480029327.XA Pending CN105247052A (zh) 2013-05-24 2014-05-26 B-细胞cll/淋巴瘤11a(bcl11a)的寡核苷酸调节剂及其用途

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Country Link
US (1) US20160130582A1 (enExample)
EP (1) EP3004352B1 (enExample)
JP (1) JP2016520310A (enExample)
KR (1) KR20160013110A (enExample)
CN (1) CN105247052A (enExample)
BR (1) BR112015029327A2 (enExample)
CA (1) CA2907694A1 (enExample)
DK (1) DK3004352T3 (enExample)
HK (1) HK1213595A1 (enExample)
MX (1) MX363846B (enExample)
RU (1) RU2015155332A (enExample)
WO (1) WO2014188001A1 (enExample)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018119349A1 (en) * 2016-12-22 2018-06-28 University Of Maryland, Baltimore Methods for generating multipotent stem cells from tonsillar biopsies

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR101881596B1 (ko) 2008-12-02 2018-07-24 웨이브 라이프 사이언시스 재팬 인코포레이티드 인 원자 변형된 핵산의 합성 방법
RU2612521C2 (ru) 2009-07-06 2017-03-09 Онтории, Инк. Новые пролекарства нуклеиновых кислот и способы их применения
JP5868324B2 (ja) 2010-09-24 2016-02-24 株式会社Wave Life Sciences Japan 不斉補助基
BR112014001244A2 (pt) 2011-07-19 2017-02-21 Wave Life Sciences Pte Ltd métodos para a síntese de ácidos nucléicos funcionalizados
CN112007045A (zh) 2012-07-13 2020-12-01 波涛生命科学有限公司 手性控制
ES2862073T3 (es) 2012-07-13 2021-10-06 Wave Life Sciences Ltd Grupo auxiliar asimétrico
EP3095459A4 (en) 2014-01-15 2017-08-23 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having antitumor effect and antitumor agent
US10144933B2 (en) 2014-01-15 2018-12-04 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having immunity induction activity, and immunity induction activator
CN113278617A (zh) 2014-01-16 2021-08-20 波涛生命科学有限公司 手性设计
WO2017184082A1 (en) * 2016-04-22 2017-10-26 Nanyang Technological University A lymphocyte permeating chimeric oligonucleotide, methods and uses thereof
TW201839136A (zh) 2017-02-06 2018-11-01 瑞士商諾華公司 治療血色素異常症之組合物及方法
EP3880819A2 (en) 2018-11-13 2021-09-22 Lipigon Pharmaceuticals AB Angptl4 oligonucleotides influencing the regulation of the fatty acid metabolism
CN114903992B (zh) * 2022-04-18 2024-01-16 南通大学 转录因子bcl11a在制备施万细胞调控药物中的应用
WO2025024334A1 (en) 2023-07-21 2025-01-30 Marrow Therapeutics, Inc. Hematopoietic cell targeting conjugates and related methods

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2010030963A2 (en) * 2008-09-15 2010-03-18 Children's Medical Center Corporation Modulation of bcl11a for treatment of hemoglobinopathies
US20100323967A1 (en) * 2007-12-07 2010-12-23 Santaris Pharma A/S RNA Antagonist Compounds for the Modulation of MCL-1
CN102329794A (zh) * 2011-09-28 2012-01-25 暨南大学 抑制BCL11A表达和肿瘤性B细胞增殖的Bcl11a siRNA-585及其应用
WO2012079046A2 (en) * 2010-12-10 2012-06-14 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of klf-1 and bcl11a genes

Family Cites Families (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6617442B1 (en) 1999-09-30 2003-09-09 Isis Pharmaceuticals, Inc. Human Rnase H1 and oligonucleotide compositions thereof
DK2284269T3 (en) 2002-11-18 2017-10-23 Roche Innovation Ct Copenhagen As Antisense design
WO2007031091A2 (en) 2005-09-15 2007-03-22 Santaris Pharma A/S Rna antagonist compounds for the modulation of p21 ras expression
EP1984381B1 (en) 2006-01-27 2010-09-29 Isis Pharmaceuticals, Inc. 6-modified bicyclic nucleic acid analogs
WO2007131237A2 (en) 2006-05-05 2007-11-15 Isis Pharmaceuticals, Inc. Compounds and methods for modulating expression of ptp1b
ES2389737T3 (es) 2006-05-11 2012-10-31 Isis Pharmaceuticals, Inc. Análogos de ácidos nucleicos bicíclicos modificados en 5'
EP2090127A2 (en) 2006-10-30 2009-08-19 Nokia Corporation Method, apparatus and system providing operator controlled mobility for user equipment
WO2008113832A2 (en) * 2007-03-22 2008-09-25 Santaris Pharma A/S SHORT RNA ANTAGONIST COMPOUNDS FOR THE MODULATION OF TARGET mRNA
US8278425B2 (en) 2007-05-30 2012-10-02 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
DK2173760T4 (en) 2007-06-08 2016-02-08 Isis Pharmaceuticals Inc Carbocyclic bicyclic nukleinsyreanaloge
US8278283B2 (en) 2007-07-05 2012-10-02 Isis Pharmaceuticals, Inc. 6-disubstituted or unsaturated bicyclic nucleic acid analogs
US8546556B2 (en) 2007-11-21 2013-10-01 Isis Pharmaceuticals, Inc Carbocyclic alpha-L-bicyclic nucleic acid analogs
WO2009090182A1 (en) 2008-01-14 2009-07-23 Santaris Pharma A/S C4'-substituted - dna nucleotide gapmer oligonucleotides
EP2265627A2 (en) 2008-02-07 2010-12-29 Isis Pharmaceuticals, Inc. Bicyclic cyclohexitol nucleic acid analogs
EP2274423A2 (en) 2008-04-04 2011-01-19 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleosides and having reduced toxicity
WO2010036698A1 (en) 2008-09-24 2010-04-01 Isis Pharmaceuticals, Inc. Substituted alpha-l-bicyclic nucleosides
EP2462153B1 (en) 2009-08-06 2015-07-29 Isis Pharmaceuticals, Inc. Bicyclic cyclohexose nucleic acid analogs
WO2011085102A1 (en) 2010-01-11 2011-07-14 Isis Pharmaceuticals, Inc. Base modified bicyclic nucleosides and oligomeric compounds prepared therefrom
WO2011115818A1 (en) 2010-03-17 2011-09-22 Isis Pharmaceuticals, Inc. 5'-substituted bicyclic nucleosides and oligomeric compounds prepared therefrom
WO2012073047A2 (en) * 2010-12-03 2012-06-07 Genome Research Limited Compositions and methods
CN102352356B (zh) * 2011-09-28 2012-12-05 暨南大学 抑制BCL11A表达和肿瘤性B细胞增殖的Bcl11a siRNA-2292及其应用
WO2013055985A1 (en) * 2011-10-12 2013-04-18 Children's Medical Center Corporation Combinatorial compositions and methods of treating hemoglobinopathies

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20100323967A1 (en) * 2007-12-07 2010-12-23 Santaris Pharma A/S RNA Antagonist Compounds for the Modulation of MCL-1
WO2010030963A2 (en) * 2008-09-15 2010-03-18 Children's Medical Center Corporation Modulation of bcl11a for treatment of hemoglobinopathies
WO2012079046A2 (en) * 2010-12-10 2012-06-14 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of klf-1 and bcl11a genes
CN102329794A (zh) * 2011-09-28 2012-01-25 暨南大学 抑制BCL11A表达和肿瘤性B细胞增殖的Bcl11a siRNA-585及其应用

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
《BIO-SYNTHESIS, INC.》 *
《NUCLEIC ACIDS RESEARCH》 *
《THE JOURNAL OF BIOLOGICAL CHEMISTRY》 *
K L A U S D . L I N S E K L A U S D . L I N S E: "What is a Gapmer?", 《BIO-SYNTHESIS, INC.》 *
杨璐 等: "2′-O-甲氧乙基反义寡核苷酸应用于杜兴肌肉萎缩症的体外研究", 《天津医科大学学报》 *

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018119349A1 (en) * 2016-12-22 2018-06-28 University Of Maryland, Baltimore Methods for generating multipotent stem cells from tonsillar biopsies
US11957718B2 (en) 2016-12-22 2024-04-16 University Of Maryland, Baltimore Methods for generating multipotent stem cells from tonsillar biopsies

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RU2015155332A (ru) 2017-06-27
HK1213595A1 (zh) 2016-07-08
DK3004352T3 (da) 2017-11-27
MX2015015564A (es) 2016-02-05
US20160130582A1 (en) 2016-05-12
KR20160013110A (ko) 2016-02-03
CA2907694A1 (en) 2014-11-27
JP2016520310A (ja) 2016-07-14
EP3004352B1 (en) 2017-09-27
EP3004352A1 (en) 2016-04-13
BR112015029327A2 (pt) 2017-09-19
MX363846B (es) 2019-04-05
WO2014188001A1 (en) 2014-11-27

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