CA2708354C - Means for delivery of nucleic acids active for gene silencing using synthetic polymers - Google Patents

Means for delivery of nucleic acids active for gene silencing using synthetic polymers Download PDF

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Publication number
CA2708354C
CA2708354C CA2708354A CA2708354A CA2708354C CA 2708354 C CA2708354 C CA 2708354C CA 2708354 A CA2708354 A CA 2708354A CA 2708354 A CA2708354 A CA 2708354A CA 2708354 C CA2708354 C CA 2708354C
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CA
Canada
Prior art keywords
sirna
tyrosine
rna
tryptophan
polyethylenimine
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Active
Application number
CA2708354A
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English (en)
French (fr)
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CA2708354A1 (en
Inventor
Abdennaji Adib
Patrick Erbacher
Fabrice Stock
Nadia Hafdi
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Polyplus Transfection SA
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Polyplus Transfection SA
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Publication of CA2708354A1 publication Critical patent/CA2708354A1/en
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0041—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Polyamides (AREA)
CA2708354A 2007-12-13 2008-12-12 Means for delivery of nucleic acids active for gene silencing using synthetic polymers Active CA2708354C (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US1335807P 2007-12-13 2007-12-13
US61/013,358 2007-12-13
PCT/IB2008/055256 WO2009074970A2 (en) 2007-12-13 2008-12-12 Means for delivery of nucleic acids active for gene silencing using synthetic polymers

Publications (2)

Publication Number Publication Date
CA2708354A1 CA2708354A1 (en) 2009-06-18
CA2708354C true CA2708354C (en) 2018-12-11

Family

ID=40561891

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2708354A Active CA2708354C (en) 2007-12-13 2008-12-12 Means for delivery of nucleic acids active for gene silencing using synthetic polymers

Country Status (8)

Country Link
US (1) US20100297756A1 (https=)
EP (1) EP2229445B1 (https=)
JP (1) JP5802389B2 (https=)
KR (1) KR101590652B1 (https=)
CN (1) CN101952434B (https=)
CA (1) CA2708354C (https=)
IL (1) IL206310A (https=)
WO (1) WO2009074970A2 (https=)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8945927B2 (en) 2010-03-29 2015-02-03 Universite De Strasbourg Polymers for delivering molecules of interest
KR101223483B1 (ko) * 2010-09-10 2013-01-17 한국과학기술연구원 전하결합 및 생분해성 공유결합으로 동시에 연결된 고분자―ｓｉＲＮＡ 나노입자 전달체
KR101497668B1 (ko) 2012-09-28 2015-03-03 한양대학교 산학협력단 전신 순환을 위한 짧은 간섭 rna 유전자 전달체
WO2014051318A2 (ko) * 2012-09-28 2014-04-03 한양대학교 산학협력단 전신 순환을 위한 짧은 간섭 rna 유전자 전달체
WO2014053245A1 (en) 2012-10-05 2014-04-10 Lipocalyx Gmbh Hydroxylated polyamine derivatives as transfection reagents
AU2013329889B2 (en) 2012-10-08 2018-03-08 Biontech Delivery Technologies Gmbh Carboxylated polyamine derivatives as transfection reagents
CN103087314B (zh) * 2013-02-01 2015-02-18 同济大学 生物可降解聚氨酯基因转染试剂的制备方法
CN107805643B (zh) * 2017-09-15 2020-12-08 四川大学 靶向抑制沙门氏菌耐药外排泵基因acrA的siRNA-DNA纳米系统及其制备方法
BR112022002143A2 (pt) * 2019-08-05 2022-04-19 Polyplus Transfection Composição adequada para transfectar uma molécula de ácido nucleico em uma célula, métodos para transfecção in vitro ou ex vivo de células vivas e para a produção e usos in vitro ou ex vivo da composição
DE102020114183B4 (de) 2020-05-27 2022-07-14 Universität Leipzig Dezernat Forschung und Transfer Mittel zur transfektion von nukleinsäuren in zellen
CN112195193A (zh) * 2020-09-30 2021-01-08 上海交通大学 单链dna在基因转染中的应用
WO2023164285A1 (en) * 2022-02-28 2023-08-31 Northwestern University DISE-INDUCING sRNA-POLYPLEXES AND sRNA-LIPOPOLYPLEXES AND METHODS OF USING THE SAME TO TREAT CANCER
WO2026036019A1 (en) 2024-08-09 2026-02-12 Life Technologies Corporation Method of producing a recombinant protein in hek-293 cells

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7427394B2 (en) * 2000-10-10 2008-09-23 Massachusetts Institute Of Technology Biodegradable poly(β-amino esters) and uses thereof
US7601367B2 (en) * 2002-05-28 2009-10-13 Mirus Bio Llc Compositions and processes using siRNA, amphipathic compounds and polycations
CN1720257A (zh) * 2002-08-06 2006-01-11 因特拉迪格姆公司 通过导入干扰rna在体内下调靶基因表达的方法
BRPI0407107A (pt) * 2003-01-31 2006-01-24 Novartis Ag Infra-regulação de gene alvo com complexos de oligoribonucleotìdeo de filamento único e polìmero de polietilenoimina (pei)
CA2523785A1 (en) * 2003-05-09 2004-11-25 University Of Pittsburgh Of The Commonwealth System Of Higher Education Small interfering rna libraries and methods of synthesis and use
ES2559828T3 (es) * 2003-07-16 2016-02-16 Protiva Biotherapeutics Inc. ARN de interferencia encapsulado en lípidos
US7842673B2 (en) * 2003-12-17 2010-11-30 The Trustees Of Columbia University In The City Of New York Delivery of DNA or RNA via gap junctions from host cells to target cells and a cell-based delivery system for antisense or siRNA
EP1841867A1 (en) * 2005-01-24 2007-10-10 Avaris AB COMPLEX CONTAINING SiRNA, ShRNA OR ANTISENSE MOLECULE AND FUNCTIONAL ENTITY, FOR IMPROVED SPECIFICITY AND DELIVERY
US20100310670A1 (en) * 2005-09-02 2010-12-09 Takeda Pharmaceutical Company Limited Sustained-release microsphere containing short chain deoxyribonucleic acid or short chain ribonucleic acid and method of producing the same
KR100863405B1 (ko) * 2005-09-12 2008-10-14 주식회사 대웅 암 진단 마커 및 이의 이용
JP2007119396A (ja) * 2005-10-28 2007-05-17 Hosokawa Funtai Gijutsu Kenkyusho:Kk 核酸化合物封入ナノ粒子を含む経肺投与用医薬製剤
GB0613753D0 (en) * 2006-07-11 2006-08-23 Norwegian Radium Hospital Res Method
CN101085356A (zh) * 2007-06-21 2007-12-12 中国科学院长春应用化学研究所 一种非病毒基因转染载体、其与质粒dna复合物颗粒及制备方法和用法

Also Published As

Publication number Publication date
CA2708354A1 (en) 2009-06-18
CN101952434A (zh) 2011-01-19
EP2229445A2 (en) 2010-09-22
IL206310A (en) 2013-07-31
US20100297756A1 (en) 2010-11-25
IL206310A0 (en) 2010-12-30
KR20100105674A (ko) 2010-09-29
JP2011505826A (ja) 2011-03-03
WO2009074970A2 (en) 2009-06-18
JP5802389B2 (ja) 2015-10-28
EP2229445B1 (en) 2012-12-05
CN101952434B (zh) 2016-11-16
WO2009074970A3 (en) 2009-10-29
KR101590652B1 (ko) 2016-02-18

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