CA2759438C - Gene vectors and uses thereof for preventing or reducing expression of a nucleotide sequence in a hematopoietic stem or progenitor cell but not in a differentiated cell - Google Patents

Gene vectors and uses thereof for preventing or reducing expression of a nucleotide sequence in a hematopoietic stem or progenitor cell but not in a differentiated cell Download PDF

Info

Publication number
CA2759438C
CA2759438C CA2759438A CA2759438A CA2759438C CA 2759438 C CA2759438 C CA 2759438C CA 2759438 A CA2759438 A CA 2759438A CA 2759438 A CA2759438 A CA 2759438A CA 2759438 C CA2759438 C CA 2759438C
Authority
CA
Canada
Prior art keywords
cell
cells
mir
expression
galc
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
CA2759438A
Other languages
English (en)
French (fr)
Other versions
CA2759438A1 (en
Inventor
Alessandra Biffi
Bernhard Rudolf Gentner
Luigi Naldini
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Fondazione Telethon
Ospedale San Raffaele SRL
Original Assignee
Fondazione Telethon
Ospedale San Raffaele SRL
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 Fondazione Telethon, Ospedale San Raffaele SRL filed Critical Fondazione Telethon
Publication of CA2759438A1 publication Critical patent/CA2759438A1/en
Application granted granted Critical
Publication of CA2759438C publication Critical patent/CA2759438C/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • 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
    • 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
    • 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
    • C12N15/63—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
    • 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
    • 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)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Diabetes (AREA)
  • Hematology (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)
CA2759438A 2009-04-30 2010-04-30 Gene vectors and uses thereof for preventing or reducing expression of a nucleotide sequence in a hematopoietic stem or progenitor cell but not in a differentiated cell Active CA2759438C (en)

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

Publications (2)

Publication Number Publication Date
CA2759438A1 CA2759438A1 (en) 2010-11-04
CA2759438C true CA2759438C (en) 2021-03-30

Family

ID=42790671

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2759438A Active CA2759438C (en) 2009-04-30 2010-04-30 Gene vectors and uses thereof for preventing or reducing expression of a nucleotide sequence in a hematopoietic stem or progenitor cell but not in a differentiated cell

Country Status (23)

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

Families Citing this family (36)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SI2424571T1 (sl) * 2009-04-30 2020-10-30 Ospedale San Raffaele S.R.L. Genski vektor
LT2772265T (lt) 2010-05-14 2018-05-25 Oregon Health & Science University Rekombinantiniai žcmv ir rhcmv vektoriai ir jų panaudojimas
WO2012006577A2 (en) * 2010-07-08 2012-01-12 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
US10266850B2 (en) 2012-05-25 2019-04-23 The Regents Of The University Of California Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
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
CA3034794A1 (en) 2012-12-06 2014-06-12 Sigma-Aldrich Co. Llc Crispr-based genome modification and regulation
RS64664B1 (sr) 2013-02-15 2023-11-30 Bioverativ Therapeutics Inc Gen optimizovanog faktora viii
DK3620534T3 (da) 2013-03-14 2021-12-06 Caribou Biosciences Inc Crispr-cas sammensætninger af nucleinsyre-targeting nucleinsyrer
CA2927712A1 (en) 2013-10-24 2015-04-30 Ospedale San Raffaele S.R.L. Method for the genetic modification of haematopoietic stem and progenitor cells (hspcs) and the use of genetically modified hspcs in gene therapy
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
WO2016201507A1 (en) * 2015-06-15 2016-12-22 Murdoch Childrens Research Institute Method of measuring chimerism
JP6990176B2 (ja) 2015-10-05 2022-02-03 モデルナティエックス インコーポレイテッド メッセンジャーリボ核酸薬物の治療投与のための方法
US10188750B1 (en) 2015-10-23 2019-01-29 University Of South Florida Self-replicating cell selective gene delivery compositions, methods, and uses thereof
PH12018501628B1 (en) 2016-02-01 2023-11-10 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
CR20190205A (es) * 2016-10-18 2020-01-07 Univ Oregon Health & Science Vectores de citomegalovirus que provocan células t restringidas por moléculas del complejo mayor de histocompatibilidad e
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
EP4491233A3 (en) * 2017-04-21 2025-04-16 Ospedale San Raffaele S.r.l. Gene therapy
IT201700086403A1 (it) * 2017-07-27 2019-01-27 Univ Del Piemonte Orientale Endothelial-specific promoter sequences and uses thereof
NZ762100A (en) 2017-08-09 2025-12-19 Bioverativ Therapeutics Inc Nucleic acid molecules and uses thereof
JP2021512126A (ja) 2018-02-01 2021-05-13 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 第ｖｉｉｉ因子を発現するレンチウイルスベクターの使用
EP3833766A1 (en) 2018-08-09 2021-06-16 Bioverativ Therapeutics Inc. Nucleic acid molecules and uses thereof for non-viral gene therapy
CN113396223A (zh) 2018-12-06 2021-09-14 比奥维拉迪维治疗股份有限公司 表达因子ix的慢病毒载体的用途
CN115279896A (zh) 2019-09-30 2022-11-01 比奥维拉迪维治疗股份有限公司 慢病毒载体配制品
US20220378937A1 (en) * 2019-11-12 2022-12-01 The Regents Of The University Of California Lentiviral vectors in hematopoietic stem cells to treat x-linked chronic granulomatous disease
IL299333A (en) 2020-06-24 2023-02-01 Bioverativ Therapeutics Inc Methods for the purification of viral vectors
CN116194152A (zh) 2020-08-23 2023-05-30 比奥维拉迪维治疗股份有限公司 用于改进封闭端DNA（ceDNA）的产生的经修饰的杆状病毒系统
EP4392566A2 (en) 2021-08-23 2024-07-03 Bioverativ Therapeutics Inc. Baculovirus expression system
AU2022332276A1 (en) 2021-08-23 2024-04-04 Bioverativ Therapeutics Inc. Optimized factor viii genes
WO2023056331A1 (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

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
KR20000029858A (ko) 1996-08-07 2000-05-25 마르크 젠너 엠엠피와티엔에프억제활성을갖는히드록삼산및카르복실산유도체
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
EP2474616B1 (en) 2004-05-28 2015-07-08 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
KR20130114758A (ko) * 2005-05-27 2013-10-17 오스페달레 산 라파엘 에스.알.엘. Ｍｉ-ｒｎａ를 포함하는 유전자 벡터
JP2008541737A (ja) * 2005-06-03 2008-11-27 サウザーン アデレード ヘルス サービス−ファインダーズ メディカル センター マイクロｒｎａ発現の変化した細胞を標的とすること
US20090180989A1 (en) * 2005-10-05 2009-07-16 Synageva Biopharma Corp. Compositions and methods for delivering nucleotide sequences to vertebrates
EP2487254B1 (en) 2006-01-05 2015-07-29 The Ohio State University Research Foundation MicroRNA-based methods and compositions for the diagnosis and treatment of solid cancers
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
EP3252161B1 (en) 2007-06-06 2021-11-17 Genzyme Corporation Gene therapy for lysosomal storage diseases
WO2010055413A1 (en) 2008-11-12 2010-05-20 Fondazione Centro San Raffaele Del Monte Tabor Gene vector for inducing transgene-specific immune tolerance
SI2424571T1 (sl) 2009-04-30 2020-10-30 Ospedale San Raffaele S.R.L. Genski vektor

Also Published As

Publication number Publication date
WO2010125471A2 (en) 2010-11-04
JP2016028582A (ja) 2016-03-03
US11407996B2 (en) 2022-08-09
CA2759438A1 (en) 2010-11-04
SG175839A1 (en) 2011-12-29
AU2010243276A1 (en) 2011-11-17
US20190367911A1 (en) 2019-12-05
US20180044670A1 (en) 2018-02-15
HUE049845T2 (hu) 2020-10-28
EA201171335A1 (ru) 2012-05-30
IL215804A (en) 2016-10-31
IL215804A0 (en) 2012-01-31
BRPI1010873B1 (pt) 2020-04-14
US10287579B2 (en) 2019-05-14
HRP20200647T1 (hr) 2020-10-02
BRPI1010873A2 (pt) 2016-03-15
WO2010125471A3 (en) 2010-12-29
JP2012525141A (ja) 2012-10-22
CY1123218T1 (el) 2021-10-29
EP2424571A2 (en) 2012-03-07
SG10201610827RA (en) 2017-02-27
EA023938B1 (ru) 2016-07-29
KR20120038403A (ko) 2012-04-23
PT2424571T (pt) 2020-05-06
KR101793615B1 (ko) 2017-11-03
SMT202000224T1 (it) 2020-05-08
CN102596255B (zh) 2017-10-13
SI2424571T1 (sl) 2020-10-30
US20220333103A1 (en) 2022-10-20
EP3733214A1 (en) 2020-11-04
US9951328B2 (en) 2018-04-24
LT2424571T (lt) 2020-08-10
MX2011011508A (es) 2012-02-13
ES2786039T3 (es) 2020-10-08
AU2010243276B2 (en) 2016-09-15
US20120128643A1 (en) 2012-05-24
CN102596255A (zh) 2012-07-18
PL2424571T3 (pl) 2020-10-19
JP6144911B2 (ja) 2017-06-07
DK2424571T3 (da) 2020-05-04
EP2424571B1 (en) 2020-04-08
JP6370278B2 (ja) 2018-08-08

Similar Documents

Publication Publication Date Title
US20220333103A1 (en) Gene vector
Wang et al. Brain endothelial cells maintain lactate homeostasis and control adult hippocampal neurogenesis
JP6713073B2 (ja) 異常ヘモグロビン症を治療するための組成物および方法
US20160348103A1 (en) Oligonucleotides and Methods for Treatment of Cardiomyopathy Using RNA Interference
US20090202493A1 (en) Mir-150 for the treatment of blood disorders
Schiffelers et al. siRNA-mediated inhibition of angiogenesis
HK40021359A (en) Compositions and methods to treating hemoglobinopathies
Chaudhuri MicroRNAs 155 and 125b physiologically and pathologically regulate hematopoiesis and immunity
HK1233490A1 (en) Compositions and methods to treating hemoglobinopathies
HK1233490B (en) Compositions and methods to treating hemoglobinopathies

Legal Events

Date Code Title Description
EEER Examination request

Effective date: 20150106