EA023938B1 - Вектор гена - Google Patents

Вектор гена Download PDF

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Publication number
EA023938B1
EA023938B1 EA201171335A EA201171335A EA023938B1 EA 023938 B1 EA023938 B1 EA 023938B1 EA 201171335 A EA201171335 A EA 201171335A EA 201171335 A EA201171335 A EA 201171335A EA 023938 B1 EA023938 B1 EA 023938B1
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EA
Eurasian Patent Office
Prior art keywords
cell
cells
mirna
expression
vector
Prior art date
Application number
EA201171335A
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English (en)
Russian (ru)
Other versions
EA201171335A1 (ru
Inventor
Алессандра Биффи
Бернхард Рудольф Гентнер
Луиджи Нальдини
Original Assignee
Оспедале Сан Рафаэлле С.Р.Л.
Фондационе Телетон
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Application filed by Оспедале Сан Рафаэлле С.Р.Л., Фондационе Телетон filed Critical Оспедале Сан Рафаэлле С.Р.Л.
Publication of EA201171335A1 publication Critical patent/EA201171335A1/ru
Publication of EA023938B1 publication Critical patent/EA023938B1/ru

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    • 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
    • C12N15/11—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

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  • 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)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Hematology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Diabetes (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)
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 EA201171335A1 (ru) 2012-05-30
EA023938B1 true EA023938B1 (ru) 2016-07-29

Family

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Family Applications (1)

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

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)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2749971C2 (ru) * 2016-09-22 2021-06-21 Юниверсити Оф Массачусетс Средство лечения болезни гентингтона на основе aav

Families Citing this family (37)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL2424571T3 (pl) * 2009-04-30 2020-10-19 Ospedale San Raffaele S.R.L Wektor genowy
TR201802741T4 (tr) 2010-05-14 2018-03-21 Univ Oregon Health & Science Rekombinant hcmv ve rhcmv vektörleri ve bunların kullanımları.
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
US20150190532A1 (en) * 2012-04-04 2015-07-09 The Trustees Of Columbia University In The City Of New York Smooth muscle specific inhibition for anti-restenotic therapy
AU2013266968B2 (en) 2012-05-25 2017-06-29 Emmanuelle CHARPENTIER 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
PL3360964T3 (pl) 2012-12-06 2020-03-31 Sigma-Aldrich Co. Llc Modyfikacja i regulacja genomu oparta na crispr
WO2014127215A1 (en) 2013-02-15 2014-08-21 Biogen Idec Ma Inc. Optimized factor viii gene
RU2662932C2 (ru) 2013-03-14 2018-07-31 Карибо Биосайенсиз, Инк. Композиции и способы с участием нуклеиновых кислот, нацеленных на нуклеиновые кислоты
EP3060670B1 (en) 2013-10-24 2019-07-10 Ospedale San Raffaele S.r.l. 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
AU2016281193B2 (en) * 2015-06-15 2022-06-16 Murdoch Childrens Research Institute Method of measuring chimerism
EP3359670B2 (en) 2015-10-05 2024-02-14 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
CA3012695A1 (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
US10532099B2 (en) * 2016-10-18 2020-01-14 Oregon Health & Science University Cytomegalovirus vectors eliciting T cells restricted by major histocompatibility complex E molecules
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
IL270028B2 (en) * 2017-04-21 2025-10-01 Ospedale San Raffaele Srl Garden care
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
AU2019215063B2 (en) 2018-02-01 2025-10-16 Bioverativ Therapeutics, Inc. Use of lentiviral vectors expressing Factor VIII
EP3833766A1 (en) 2018-08-09 2021-06-16 Bioverativ Therapeutics Inc. Nucleic acid molecules and uses thereof for non-viral gene therapy
JP7644007B2 (ja) 2018-12-06 2025-03-11 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ｉｘ因子を発現するレンチウイルスベクターの使用
KR20220097891A (ko) 2019-09-30 2022-07-08 바이오버라티브 테라퓨틱스 인크. 렌티바이러스 벡터 제형
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.
WO2022046665A1 (en) 2020-08-23 2022-03-03 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
MX2024002333A (es) 2021-08-23 2024-03-07 Bioverativ Therapeutics Inc Sistema de expresion de baculovirus.
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
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
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

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2006128245A1 (en) * 2005-06-03 2006-12-07 Southern Adelaide Health Service-Flinders Medical Centre Targeting cells with altered microrna expression

Family Cites Families (14)

* 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
WO1998005635A1 (en) 1996-08-07 1998-02-12 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
RU2305708C2 (ru) * 2001-10-02 2007-09-10 Энститю Клейтон Де Ля Решерш Рекомбинантный лентивирусный вектор, клетка-хозяин, трансдуцированная лентивирусным вектором, способ ее трансдукции и применение
EP2290073A3 (en) 2004-05-28 2011-08-31 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
CA2609142C (en) * 2005-05-27 2016-02-09 Fondazione Centro San Raffaele Del Monte Tabor Therapeutic gene vectors comprising mirna target sequences
US20090180989A1 (en) * 2005-10-05 2009-07-16 Synageva Biopharma Corp. Compositions and methods for delivering nucleotide sequences to vertebrates
EP1969147B1 (en) 2006-01-05 2014-07-30 The Ohio State University Research Foundation microRNA-based methods for the diagnosis of 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
EP2104736B1 (en) 2006-12-08 2011-11-23 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
PL2424571T3 (pl) 2009-04-30 2020-10-19 Ospedale San Raffaele S.R.L Wektor genowy

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2006128245A1 (en) * 2005-06-03 2006-12-07 Southern Adelaide Health Service-Flinders Medical Centre Targeting cells with altered microrna expression

Non-Patent Citations (8)

* Cited by examiner, † Cited by third party
Title
BROWN BRIAN D. ET AL.: "Endogenous microRNA can be broadly exploited to regulate transgene expression according to tissue, lineage and differentiation state". NATURE BIOTECHNOLOGY, vol. 25, no. 12, 16 November 2007 (2007-11-16), pages 1457-1467, XP002471752, ISSN: 1087-0156, cited in the application, the whole document *
CARTIER NATHALIE ET AL.: "Hematopoietic stem cell gene therapy in Hurler syndrome, globoid cell leukodystrophy, metachromatic leukodystrophy and X-adrenoleukodystrophy". CURRENT OPINION IN MOLECULAR THERAPEUTICS, vol. 10, no. 5, October 2008 (2008-10), pages 471-478, XP008127749, ISSN: 1464-8431, the whole document *
GARZON RAMIRO ET AL.: "MicroRNA fingerprints during human megakaryocytopoiesis". PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES, vol. 103, no. 13, 28 March 2006 (2006-03-28), pages 5078-5083, XP002465978, ISSN: 0027-8424, the whole document *
GENTNER BERNHARD ET AL.: "Characterization of Hematopoietic Stem Cell Specific microRNA: From Physiologic Function to Gene Therapy Applications". MOLECULAR THERAPY, vol. 17, no. Suppl. 1, May 2009 (2009-05), page S20, XP008127618, ISSN: 1525-0016, the whole document & 12TH ANNUAL MEETING OF THE AMERICAN SOCIETY OF GENE THERAPY; SAN DIEGO, CA, USA; MAY 27-30, 2009 *
GENTNER ET AL.: "Exploiting microRNA expression profiles for lineage- and differentiation state-specific transgene expression in hematopoietic, neural and embryonic stem cells". BLOOD CELLS, MOLECULES AND DISEASES, vol. 40, no. 2, 12 February 2008 (2008-02-12), page 267, XP022477840, ISSN: 1079-9796, the whole document *
LECHMAN ERIC R. ET AL.: "High Levels of MicroRNA-126 Bioactivity Specify the LSC Compartment in AML" BLOOD, vol. 112, no. 11, November 2008 (2008-11), page 193, XP008127737, ISSN: 0006-4971, the whole document & 50TH ANNUAL MEETING OF THE AMERICAN-SOCIETY-OF-HEMATOLOGY; SAN FRANCISCO, CA, USA; DECEMBER 06-09, 2008 *
PAPAPETROU EIRINI P. ET AL.: "MicroRNA-mediated gene regulation effectively restricts in vivo transgene expression in hematopoietic stem cell progeny", BLOOD, vol. 110, no. 11, Part 1, November 2007 (2007-11), pages 64A-65A, XP008127620, ISSN: 0006-4971, the whole document & 49TH ANNUAL MEETING OF THE AMERICAN-SOCIETY-OF-HEMATOLOGY; ATLANTA, GA, USA; DECEMBER 08-11, 2007 *
RYSER MARTIN F. ET AL.: "Gene therapy for chronic granulomatous disease". EXPERT OPINION ON BIOLOGICAL THERAPY, vol. 7, no. 12, December 2007 (2007-12), pages 1799-1809, XP008127764, ISSN: 1744-7682, the whole document *

Cited By (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2749971C2 (ru) * 2016-09-22 2021-06-21 Юниверсити Оф Массачусетс Средство лечения болезни гентингтона на основе aav
US11046957B2 (en) 2016-09-22 2021-06-29 University Of Massachusetts AAV treatment of Huntington's disease
US11773392B2 (en) 2016-09-22 2023-10-03 University Of Massachusetts AAV treatment of Huntington's disease
US12522824B2 (en) 2016-09-22 2026-01-13 University Of Massachusetts AAV treatment of Huntington's disease

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Publication number Publication date
CN102596255A (zh) 2012-07-18
US20120128643A1 (en) 2012-05-24
IL215804A (en) 2016-10-31
EA201171335A1 (ru) 2012-05-30
US20180044670A1 (en) 2018-02-15
ES2786039T3 (es) 2020-10-08
LT2424571T (lt) 2020-08-10
JP6370278B2 (ja) 2018-08-08
EP2424571B1 (en) 2020-04-08
WO2010125471A3 (en) 2010-12-29
AU2010243276B2 (en) 2016-09-15
SG175839A1 (en) 2011-12-29
BRPI1010873B1 (pt) 2020-04-14
MX2011011508A (es) 2012-02-13
SG10201610827RA (en) 2017-02-27
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