EA201171335A1 - Вектор гена - Google Patents

Вектор гена

Info

Publication number
EA201171335A1
EA201171335A1 EA201171335A EA201171335A EA201171335A1 EA 201171335 A1 EA201171335 A1 EA 201171335A1 EA 201171335 A EA201171335 A EA 201171335A EA 201171335 A EA201171335 A EA 201171335A EA 201171335 A1 EA201171335 A1 EA 201171335A1
Authority
EA
Eurasian Patent Office
Prior art keywords
hsc
nucleotide sequence
vector gene
cell
mirna
Prior art date
Application number
EA201171335A
Other languages
English (en)
Other versions
EA023938B1 (ru
Inventor
Алессандра Биффи
Бернхард Рудольф Гентнер
Луиджи Нальдини
Original Assignee
Фондационе Чентро Сан Рафаэле Дель Монтэ Табор
Фондационе Телетон
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Фондационе Чентро Сан Рафаэле Дель Монтэ Табор, Фондационе Телетон filed Critical Фондационе Чентро Сан Рафаэле Дель Монтэ Табор
Publication of EA201171335A1 publication Critical patent/EA201171335A1/ru
Publication of EA023938B1 publication Critical patent/EA023938B1/ru

Links

Classifications

    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/04—Immunostimulants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/52—Cytokines; Lymphokines; Interferons
    • C07K14/555—Interferons [IFN]
    • C07K14/56—IFN-alpha
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • 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
    • 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 N.A.
    • C12N2310/141—MicroRNAs, miRNAs
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/51—Physical structure in polymeric form, e.g. multimers, concatemers
    • 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
    • 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
    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted vectors
    • 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
    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/027—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a retrovirus

Abstract

Вектор гена для использования в генной терапии, содержащий по меньшей мере одну мишень последовательности микроРНК, функционально связанную с нуклеотидной последовательностью, имеющей соответствующую микроРНК в гематопоэтической клетке-предшественнике (ГКП) или гематопоэтической стволовой клетке (ГСК), которые предотвращают или уменьшают экспрессию нуклеотидной последовательности в URG или ГСК, но не в дифференцированной клетке.
EA201171335A 2009-04-30 2010-04-30 Вектор гена EA023938B1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US17412409P 2009-04-30 2009-04-30
PCT/IB2010/001166 WO2010125471A2 (en) 2009-04-30 2010-04-30 Gene vector

Publications (2)

Publication Number Publication Date
EA201171335A1 true EA201171335A1 (ru) 2012-05-30
EA023938B1 EA023938B1 (ru) 2016-07-29

Family

ID=42790671

Family Applications (1)

Application Number Title Priority Date Filing Date
EA201171335A EA023938B1 (ru) 2009-04-30 2010-04-30 Вектор гена

Country Status (22)

Country Link
US (4) US10287579B2 (ru)
EP (2) EP3733214A1 (ru)
JP (2) JP6144911B2 (ru)
KR (1) KR101793615B1 (ru)
CN (1) CN102596255B (ru)
AU (1) AU2010243276B2 (ru)
BR (1) BRPI1010873B1 (ru)
CA (1) CA2759438C (ru)
CY (1) CY1123218T1 (ru)
DK (1) DK2424571T3 (ru)
EA (1) EA023938B1 (ru)
ES (1) ES2786039T3 (ru)
HR (1) HRP20200647T1 (ru)
HU (1) HUE049845T2 (ru)
IL (1) IL215804A (ru)
LT (1) LT2424571T (ru)
MX (1) MX2011011508A (ru)
PL (1) PL2424571T3 (ru)
PT (1) PT2424571T (ru)
SG (2) SG10201610827RA (ru)
SI (1) SI2424571T1 (ru)
WO (1) WO2010125471A2 (ru)

Families Citing this family (34)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2010243276B2 (en) 2009-04-30 2016-09-15 Fondazione Telethon Ets Gene vector
SI2772265T1 (en) 2010-05-14 2018-06-29 Oregon Health & Science University HCMV AND RHCMV RECOMBINANT VECTORS AND THEIR APPLICATIONS
CA2804791C (en) * 2010-07-08 2019-07-30 Duke University Direct reprogramming of cells to cardiac myocyte fate
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
WO2013152230A1 (en) * 2012-04-04 2013-10-10 The Trustees Of Columbia University In The City Of New York Smooth muscle specific inhibition for anti-restenotic therapy
JP6343605B2 (ja) 2012-05-25 2018-06-13 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア Ｒｎａ依存性標的ｄｎａ修飾およびｒｎａ依存性転写調節のための方法および組成物
JP6329537B2 (ja) 2012-07-11 2018-05-23 サンガモ セラピューティクス， インコーポレイテッド 生物学的薬剤の送達のための方法および組成物
ES2769310T3 (es) 2012-12-06 2020-06-25 Sigma Aldrich Co Llc Modificación y regulación del genoma basada en CRISPR
EP4223772A3 (en) 2013-02-15 2023-10-18 Bioverativ Therapeutics Inc. Optimized factor viii gene
CA2905432C (en) 2013-03-14 2021-09-14 Caribou Biosciences, Inc. Compositions and methods of nucleic acid-targeting nucleic acids
SG11201603166UA (en) 2013-10-24 2016-05-30 Ospedale San Raffaele Srl Method
US11008561B2 (en) 2014-06-30 2021-05-18 Bioverativ Therapeutics Inc. Optimized factor IX gene
US10501811B2 (en) 2015-04-22 2019-12-10 Kyoto University Method for sorting tissue cells
CN107960107A (zh) * 2015-06-15 2018-04-24 默多克儿童研究所 测量嵌合状态的方法
CA3001003A1 (en) 2015-10-05 2017-04-13 Modernatx, Inc. Methods for therapeutic administration of messenger ribonucleic acid drugs
US10188750B1 (en) 2015-10-23 2019-01-29 University Of South Florida Self-replicating cell selective gene delivery compositions, methods, and uses thereof
BR112018015659A2 (pt) 2016-02-01 2018-12-26 Bioverativ Therapeutics Inc genes otimizados do fator viii
US10457940B2 (en) 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
JP2019531745A (ja) 2016-10-18 2019-11-07 オレゴン ヘルス アンド サイエンス ユニバーシティ 主要組織適合遺伝子複合体ｅ分子によって拘束されるｔ細胞を誘発するサイトメガロウイルスベクター
CN106566838B (zh) * 2016-11-14 2019-11-01 上海伯豪生物技术有限公司 一种基于CRISPR-Cas9技术的miR-126全长基因敲除试剂盒及其应用
CN110785488A (zh) * 2017-04-21 2020-02-11 圣拉斐尔医院有限责任公司 基因疗法
IT201700086403A1 (it) * 2017-07-27 2019-01-27 Univ Del Piemonte Orientale Endothelial-specific promoter sequences and uses thereof
JP7374883B2 (ja) 2017-08-09 2023-11-07 バイオベラティブ セラピューティクス インコーポレイテッド 核酸分子およびその使用
CN111918674A (zh) 2018-02-01 2020-11-10 比奥维拉迪维治疗股份有限公司 表达因子viii的慢病毒载体的用途
JP2021533753A (ja) 2018-08-09 2021-12-09 バイオベラティブ セラピューティクス インコーポレイテッド 核酸分子及び非ウイルス遺伝子治療のためのそれらの使用
CA3121786A1 (en) 2018-12-06 2020-06-11 Bioverativ Therapeutics Inc. Use of lentiviral vectors expressing factor ix
EP4038182A1 (en) 2019-09-30 2022-08-10 Bioverativ Therapeutics Inc. Lentiviral vector formulations
JP2023502593A (ja) * 2019-11-12 2023-01-25 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア Ｘ連鎖慢性肉芽腫症を治療するための、造血幹細胞中のレンチウイルスベクター
MX2023000156A (es) 2020-06-24 2023-02-16 Bioverativ Therapeutics Inc Metodos para la eliminacion de factor viii libre de preparaciones de vectores lentivirales modificados para expresar dicha proteina.
US20220090130A1 (en) 2020-08-23 2022-03-24 Bioverativ Therapeutics Inc. MODIFIED BACULOVIRUS SYSTEM FOR IMPROVED PRODUCTION OF CLOSED-ENDED DNA (ceDNA)
AU2022332276A1 (en) 2021-08-23 2024-04-04 Bioverativ Therapeutics Inc. Optimized factor viii genes
CA3229668A1 (en) 2021-08-23 2023-03-02 Bioverativ Therapeutics Inc. Baculovirus expression system
TW202346327A (zh) 2021-09-30 2023-12-01 美商百歐維拉提夫治療公司 編碼具有降低免疫原性的因子ｖｉｉｉ多肽之核酸
WO2024026257A2 (en) * 2022-07-25 2024-02-01 Modernatx, Inc. Engineered polynucleotides for cell selective expression

Family Cites Families (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5723315A (en) 1996-08-23 1998-03-03 Genetics Institute, Inc. Secreted proteins and polynucleotides encoding them
EP0968182B1 (en) 1996-08-07 2004-05-06 Darwin Discovery Limited Hydroxamic and carboxylic acid derivatives having mmp and tnf inhibitory activity
US6126939A (en) 1996-09-03 2000-10-03 Yeda Research And Development Co. Ltd. Anti-inflammatory dipeptide and pharmaceutical composition thereof
NZ532060A (en) * 2001-10-02 2005-11-25 Inst Clayton De La Rech Methods and compositions relating to restricted expression lentiviral vectors and their applications
EP2471922A1 (en) 2004-05-28 2012-07-04 Asuragen, Inc. Methods and compositions involving microRNA
US20060134784A1 (en) * 2004-11-30 2006-06-22 Basch Ross S Methods and compositions for the growth and maintenance of stem cells
CA2609142C (en) * 2005-05-27 2016-02-09 Fondazione Centro San Raffaele Del Monte Tabor Therapeutic gene vectors comprising mirna target sequences
WO2006128245A1 (en) * 2005-06-03 2006-12-07 Southern Adelaide Health Service-Flinders Medical Centre Targeting cells with altered microrna expression
US20090180989A1 (en) * 2005-10-05 2009-07-16 Synageva Biopharma Corp. Compositions and methods for delivering nucleotide sequences to vertebrates
ES2536423T3 (es) 2006-01-05 2015-05-25 The Ohio State University Research Foundation Métodos basados en microARN para el diagnóstico de cáncer de próstata
US20080095750A1 (en) 2006-05-10 2008-04-24 Pennington Biomedical Research Center, Louisiana State University System Use of adipose-derived stem cells for treatment of leukodystrophies
WO2008073921A2 (en) 2006-12-08 2008-06-19 Asuragen, Inc. Mir-126 regulated genes and pathways as targets for therapeutic intervention
ES2635726T3 (es) 2007-06-06 2017-10-04 Genzyme Corporation Terapia génica para enfermedades de almacenamiento lisosómico
EP2364362B1 (en) 2008-11-12 2015-10-21 Ospedale San Raffaele S.r.l. Gene vector for inducing transgene-specific immune tolerance
AU2010243276B2 (en) * 2009-04-30 2016-09-15 Fondazione Telethon Ets Gene vector

Also Published As

Publication number Publication date
US9951328B2 (en) 2018-04-24
PT2424571T (pt) 2020-05-06
BRPI1010873A2 (pt) 2016-03-15
CN102596255B (zh) 2017-10-13
SI2424571T1 (sl) 2020-10-30
LT2424571T (lt) 2020-08-10
WO2010125471A2 (en) 2010-11-04
CN102596255A (zh) 2012-07-18
EP2424571A2 (en) 2012-03-07
HUE049845T2 (hu) 2020-10-28
EA023938B1 (ru) 2016-07-29
US20190367911A1 (en) 2019-12-05
PL2424571T3 (pl) 2020-10-19
MX2011011508A (es) 2012-02-13
JP6144911B2 (ja) 2017-06-07
CY1123218T1 (el) 2021-10-29
JP2016028582A (ja) 2016-03-03
US20220333103A1 (en) 2022-10-20
WO2010125471A3 (en) 2010-12-29
BRPI1010873B1 (pt) 2020-04-14
IL215804A0 (en) 2012-01-31
AU2010243276B2 (en) 2016-09-15
JP2012525141A (ja) 2012-10-22
KR101793615B1 (ko) 2017-11-03
DK2424571T3 (da) 2020-05-04
KR20120038403A (ko) 2012-04-23
EP2424571B1 (en) 2020-04-08
CA2759438A1 (en) 2010-11-04
JP6370278B2 (ja) 2018-08-08
EP3733214A1 (en) 2020-11-04
ES2786039T3 (es) 2020-10-08
AU2010243276A1 (en) 2011-11-17
SG10201610827RA (en) 2017-02-27
HRP20200647T1 (hr) 2020-10-02
IL215804A (en) 2016-10-31
US11407996B2 (en) 2022-08-09
US20180044670A1 (en) 2018-02-15
US10287579B2 (en) 2019-05-14
CA2759438C (en) 2021-03-30
US20120128643A1 (en) 2012-05-24
SG175839A1 (en) 2011-12-29

Similar Documents

Publication Publication Date Title
EA201171335A1 (ru) Вектор гена
MX354267B (es) Nucléosidos, nucleótidos, y ácidos nucleicos modificados, y usos de los mismos.
MX356625B (es) Oligonucleotidos para elaborar un cambio en la secuencia de una molécula de ácido ribonucleico objetivo presente en una célula viva.
MX2019008428A (es) Inhibidores de glucosilceramida sintasa.
MX2013014544A (es) Vectorales de terapia genica para adrenoleucodistrofia y adrenomieloneuropatia.
EP4269584A3 (en) Selective antisense compounds and uses thereof
NZ630596A (en) Methods for treatment of alport syndrome
MX350966B (es) Celulas madre pluripotentes.
PH12015501953A1 (en) Substituted nucleotide analogs
EP3698631A3 (en) Methods of delivering oligonucleotides to immune cells
SG196784A1 (en) Stem cell cultures
BR112015005227A2 (pt) inibidores de glicosilceramida sintase
IN2015DN03795A (ru)
WO2011159684A3 (en) Generation of induced pluripotent stem cells from small volumes of peripheral blood
WO2011130402A3 (en) Hepatocyte production by forward programming
MX2012008738A (es) Microorganismos y metodos para la biosintesis de p-toluato y tereftalato.
MX2010000348A (es) Cultivo de celulas madre pluripotentes individuales.
TN2014000327A1 (en) Oligonucleotides for modulating gene expression and uses thereof
SG10201804952QA (en) Glucosylceramide synthase inhibitors
MX2014003094A (es) Metodos para promover diferenciacion.
NZ601733A (en) Antibody with binding specificity for Robo1 protein for inhibiting the infectivity of HIV
WO2014012115A3 (en) Method for inducing cells to less mature state
WO2012127164A3 (fr) Cellule electrochimique symetrique
WO2011103028A3 (en) Compositions and methods for inhibiting mmset
MY161163A (en) Culture medium for human mesenchymal stem cells

Legal Events

Date Code Title Description
MM4A Lapse of a eurasian patent due to non-payment of renewal fees within the time limit in the following designated state(s)

Designated state(s): AM AZ BY KZ KG MD TJ TM