JP6144911B2 - 遺伝子ベクター - Google Patents

遺伝子ベクター Download PDF

Info

Publication number
JP6144911B2
JP6144911B2 JP2012507847A JP2012507847A JP6144911B2 JP 6144911 B2 JP6144911 B2 JP 6144911B2 JP 2012507847 A JP2012507847 A JP 2012507847A JP 2012507847 A JP2012507847 A JP 2012507847A JP 6144911 B2 JP6144911 B2 JP 6144911B2
Authority
JP
Japan
Prior art keywords
cells
mir
expression
galc
cell
Prior art date
Legal status (The legal status 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 status listed.)
Active
Application number
JP2012507847A
Other languages
English (en)
Japanese (ja)
Other versions
JP2012525141A5 (https=
JP2012525141A (ja
Inventor
ビッフィ，アレッサンドラ
ジェントナー，ベルンハルド，ルドルフ
ナルディーニ，ルイジ
Original Assignee
オスペダーレ サン ラファエレ エス．アール．エル
オスペダーレ サン ラファエレ エス．アール．エル
フォンダツィオーネ テレトン
フォンダツィオーネ テレトン
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 オスペダーレ サン ラファエレ エス．アール．エル, オスペダーレ サン ラファエレ エス．アール．エル, フォンダツィオーネ テレトン, フォンダツィオーネ テレトン filed Critical オスペダーレ サン ラファエレ エス．アール．エル
Publication of JP2012525141A publication Critical patent/JP2012525141A/ja
Publication of JP2012525141A5 publication Critical patent/JP2012525141A5/ja
Application granted granted Critical
Publication of JP6144911B2 publication Critical patent/JP6144911B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

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 nucleic acids [NA]
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Toxicology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
JP2012507847A 2009-04-30 2010-04-30 遺伝子ベクター Active JP6144911B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US17412409P 2009-04-30 2009-04-30
US61/174,124 2009-04-30
PCT/IB2010/001166 WO2010125471A2 (en) 2009-04-30 2010-04-30 Gene vector

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2015175986A Division JP6370278B2 (ja) 2009-04-30 2015-09-07 遺伝子ベクター

Publications (3)

Publication Number Publication Date
JP2012525141A JP2012525141A (ja) 2012-10-22
JP2012525141A5 JP2012525141A5 (https=) 2013-11-14
JP6144911B2 true JP6144911B2 (ja) 2017-06-07

Family

ID=42790671

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2012507847A Active JP6144911B2 (ja) 2009-04-30 2010-04-30 遺伝子ベクター
JP2015175986A Active JP6370278B2 (ja) 2009-04-30 2015-09-07 遺伝子ベクター

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2015175986A Active JP6370278B2 (ja) 2009-04-30 2015-09-07 遺伝子ベクター

Country Status (23)

Country Link
US (4) US10287579B2 (https=)
EP (2) EP2424571B1 (https=)
JP (2) JP6144911B2 (https=)
KR (1) KR101793615B1 (https=)
CN (1) CN102596255B (https=)
AU (1) AU2010243276B2 (https=)
BR (1) BRPI1010873B1 (https=)
CA (1) CA2759438C (https=)
CY (1) CY1123218T1 (https=)
DK (1) DK2424571T3 (https=)
EA (1) EA023938B1 (https=)
ES (1) ES2786039T3 (https=)
HR (1) HRP20200647T1 (https=)
HU (1) HUE049845T2 (https=)
IL (1) IL215804A (https=)
LT (1) LT2424571T (https=)
MX (1) MX2011011508A (https=)
PL (1) PL2424571T3 (https=)
PT (1) PT2424571T (https=)
SG (2) SG10201610827RA (https=)
SI (1) SI2424571T1 (https=)
SM (1) SMT202000224T1 (https=)
WO (1) WO2010125471A2 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016028582A (ja) * 2009-04-30 2016-03-03 オスペダーレ サン ラファエレ エス．アール．エル 遺伝子ベクター

Families Citing this family (37)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PT3351636T (pt) 2010-05-14 2020-09-24 Univ Oregon Health & Science Vectores de hcmv e rhcmv recombinantes codificadores de um antigénio heterólogo isolado a partir de um vírus paramyxoviridae e seus usos
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
EP3241902B1 (en) 2012-05-25 2018-02-28 The Regents of The University of California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
WO2014011901A2 (en) * 2012-07-11 2014-01-16 Sangamo Biosciences, Inc. Methods and compositions for delivery of biologics
EP3363902B1 (en) 2012-12-06 2019-11-27 Sigma Aldrich Co. LLC Crispr-based genome modification and regulation
SI3889173T1 (sl) 2013-02-15 2023-11-30 Bioverativ Therapeutics Inc. Optimiran gen dejavnika VIII
MX374090B (es) 2013-03-14 2025-03-05 Caribou Biosciences Inc Composiciones y métodos de ácidos nucleicos dirigidos al ácido nucleico.
ES2747951T3 (es) 2013-10-24 2020-03-12 Ospedale San Raffaele Srl Método
EP3160478A4 (en) 2014-06-30 2018-05-16 Bioverativ Therapeutics Inc. Optimized factor ix gene
JP6960159B6 (ja) * 2015-04-22 2022-06-24 国立大学法人京都大学 組織細胞の選別方法
US20180327820A1 (en) * 2015-06-15 2018-11-15 Murdoch Childrens Research Institute Method of measuring chimerism
AU2016336344A1 (en) 2015-10-05 2018-04-19 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
WO2017136358A1 (en) 2016-02-01 2017-08-10 Bioverativ Therapeutics Inc. Optimized factor viii genes
GB201612214D0 (en) 2016-07-14 2016-08-31 Univ Oxford Innovation Ltd Method
US10457940B2 (en) * 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
CN110036112B (zh) 2016-10-18 2024-05-10 俄勒冈健康与科学大学 引发受主要组织相容性复合体e分子限制的t细胞的巨细胞病毒载体
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
PT3612624T (pt) * 2017-04-21 2024-11-29 Ospedale San Raffaele Srl Terapia génica
IT201700086403A1 (it) * 2017-07-27 2019-01-27 Univ Del Piemonte Orientale Endothelial-specific promoter sequences and uses thereof
TW202545984A (zh) 2017-08-09 2025-12-01 美商生物化學醫療公司 核酸分子及其用途
AU2019215063B2 (en) 2018-02-01 2025-10-16 Bioverativ Therapeutics, Inc. Use of lentiviral vectors expressing Factor VIII
WO2020033863A1 (en) 2018-08-09 2020-02-13 Bioverativ Therapeutics Inc. Nucleic acid molecules and uses thereof for non-viral gene therapy
AU2019393880A1 (en) 2018-12-06 2021-07-15 Bioverativ Therapeutics Inc. Use of lentiviral vectors expressing factor IX
JP7803014B2 (ja) 2019-09-30 2026-01-21 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 レンチウイルスベクター製剤
AU2020384286A1 (en) * 2019-11-12 2022-06-09 The Regents Of The University Of California Lentiviral vectors in hematopoietic stem cells to treat X-linked chronic granulomatous disease
US12173307B2 (en) 2020-06-24 2024-12-24 Bioverativ Therapeutics Inc. Methods for the purification of viral vectors
US20220090130A1 (en) 2020-08-23 2022-03-24 Bioverativ Therapeutics Inc. MODIFIED BACULOVIRUS SYSTEM FOR IMPROVED PRODUCTION OF CLOSED-ENDED DNA (ceDNA)
CA3229323A1 (en) 2021-08-23 2023-03-02 Ajay MAGHODIA Optimized factor viii genes
AU2022334711A1 (en) 2021-08-23 2024-04-04 Bioverativ Therapeutics Inc. Baculovirus expression system
CN115725656B (zh) * 2021-09-01 2026-01-16 联邦生物科技(珠海横琴)有限公司 一种以细胞质rna病毒为载体递送干扰rna的方法
CA3232988A1 (en) 2021-09-30 2023-04-06 Bioverativ Therapeutics Inc. Nucleic acids encoding factor viii polypeptides with reduced immunogenicity
MA71632A (fr) * 2022-07-25 2025-05-30 Modernatx, Inc. Polynucléotides modifiés pour l'expression sélective de cellules
WO2025122739A1 (en) * 2023-12-05 2025-06-12 University Of Pittsburgh-Of The Commonwealth System Of Higher Education Compositions and methods for treating a heart disease
WO2026073244A1 (en) 2024-09-30 2026-04-02 Ensoma, Inc. Constructs for multi-lineage expression of therapeutic agents

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
DE69729007T2 (de) 1996-08-07 2005-04-07 Darwin Discovery Ltd., Slough Hydroxamsäure- und carbonsäure-derivate mit mmp und tnf hemmender wirkung
US6126939A (en) 1996-09-03 2000-10-03 Yeda Research And Development Co. Ltd. Anti-inflammatory dipeptide and pharmaceutical composition thereof
EP1438075A4 (en) * 2001-10-02 2006-04-19 Inst Clayton De La Rech METHOD AND COMPOSITIONS ASSOCIATED WITH LENTIVIRAL VECTORS WITH LIMITED EXPRESSION AND ITS APPLICATIONS
EP2290071B1 (en) 2004-05-28 2014-12-31 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
PL2002003T3 (pl) * 2005-05-27 2016-06-30 Ospedale San Raffaele Srl Wektor genetyczny zawierający mi-RNA
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
EP2487258B1 (en) 2006-01-05 2014-10-01 The Ohio State University Research Foundation MicroRNA-based methods for the diagnosis of colon, pancreas and stomach cancer
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
CA2671296A1 (en) 2006-12-08 2008-06-19 Asuragen, Inc. Mir-126 regulated genes and pathways as targets for therapeutic intervention
PL3252161T3 (pl) 2007-06-06 2022-03-07 Genzyme Corporation Terapia genowa dla lizosomalnych chorób spichrzeniowych
WO2010055413A1 (en) 2008-11-12 2010-05-20 Fondazione Centro San Raffaele Del Monte Tabor Gene vector for inducing transgene-specific immune tolerance
LT2424571T (lt) 2009-04-30 2020-08-10 Ospedale San Raffaele S.R.L. Genų vektorius

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016028582A (ja) * 2009-04-30 2016-03-03 オスペダーレ サン ラファエレ エス．アール．エル 遺伝子ベクター

Also Published As

Publication number Publication date
JP2016028582A (ja) 2016-03-03
HRP20200647T1 (hr) 2020-10-02
SMT202000224T1 (it) 2020-05-08
EP2424571A2 (en) 2012-03-07
EP2424571B1 (en) 2020-04-08
HUE049845T2 (hu) 2020-10-28
EA201171335A1 (ru) 2012-05-30
AU2010243276A1 (en) 2011-11-17
SI2424571T1 (sl) 2020-10-30
KR20120038403A (ko) 2012-04-23
EP3733214A1 (en) 2020-11-04
CN102596255B (zh) 2017-10-13
IL215804A0 (en) 2012-01-31
BRPI1010873A2 (pt) 2016-03-15
WO2010125471A2 (en) 2010-11-04
US20190367911A1 (en) 2019-12-05
WO2010125471A3 (en) 2010-12-29
JP6370278B2 (ja) 2018-08-08
CA2759438A1 (en) 2010-11-04
EA023938B1 (ru) 2016-07-29
DK2424571T3 (da) 2020-05-04
CY1123218T1 (el) 2021-10-29
IL215804A (en) 2016-10-31
KR101793615B1 (ko) 2017-11-03
SG10201610827RA (en) 2017-02-27
JP2012525141A (ja) 2012-10-22
LT2424571T (lt) 2020-08-10
CA2759438C (en) 2021-03-30
US10287579B2 (en) 2019-05-14
US20220333103A1 (en) 2022-10-20
BRPI1010873B1 (pt) 2020-04-14
PL2424571T3 (pl) 2020-10-19
SG175839A1 (en) 2011-12-29
ES2786039T3 (es) 2020-10-08
AU2010243276B2 (en) 2016-09-15
MX2011011508A (es) 2012-02-13
US20120128643A1 (en) 2012-05-24
US9951328B2 (en) 2018-04-24
US20180044670A1 (en) 2018-02-15
US11407996B2 (en) 2022-08-09
US12577560B2 (en) 2026-03-17
PT2424571T (pt) 2020-05-06
CN102596255A (zh) 2012-07-18

Similar Documents

Publication Publication Date Title
JP6370278B2 (ja) 遺伝子ベクター
Wang et al. Brain endothelial cells maintain lactate homeostasis and control adult hippocampal neurogenesis
CN111448318B (zh) 修饰在真核细胞中用于沉默基因表达的非编码rna分子的特异性的方法
EP3598984B1 (en) Compositions and methods to treating hemoglobinopathies
WO2010151755A2 (en) TREATMENT OF INFLAMMATORY DISEASES USING miR-124
CN118525089A (zh) 年龄相关的白质损失的再生治疗
US20100136026A1 (en) Ship Inhibition to Direct Hematopoietic Stem Cells and Induce Extramedullary Hematopoiesis
US10897880B2 (en) Humanized heart muscle
Chesnokova et al. Regulatory Elements for Gene Therapy of Epilepsy
JP2026505771A (ja) グリア細胞の競合的置換
WO2025181336A1 (en) Compositions and methods for hbb-editing in hspc
JP2025532585A (ja) 組み合わせ遺伝子摂動を有する免疫細胞
WO2023220364A2 (en) Improved methods and compositions for transgene delivery and/or reconstituting microglia

Legal Events

Date Code Title Description
A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20130417

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20130927

A711 Notification of change in applicant

Free format text: JAPANESE INTERMEDIATE CODE: A711

Effective date: 20140731

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20140731

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20140916

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20141216

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20150507

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20150907

A911 Transfer to examiner for re-examination before appeal (zenchi)

Free format text: JAPANESE INTERMEDIATE CODE: A911

Effective date: 20150914

A912 Re-examination (zenchi) completed and case transferred to appeal board

Free format text: JAPANESE INTERMEDIATE CODE: A912

Effective date: 20151023

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20170228

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20170512

R150 Certificate of patent or registration of utility model

Ref document number: 6144911

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250