CY1123218T1 - Διαβιβαστης γονιδιου - Google Patents

Διαβιβαστης γονιδιου

Info

Publication number
CY1123218T1
CY1123218T1 CY20201100405T CY201100405T CY1123218T1 CY 1123218 T1 CY1123218 T1 CY 1123218T1 CY 20201100405 T CY20201100405 T CY 20201100405T CY 201100405 T CY201100405 T CY 201100405T CY 1123218 T1 CY1123218 T1 CY 1123218T1
Authority
CY
Cyprus
Prior art keywords
gene transfer
hspc
hsc
nucleotide sequence
cell
Prior art date
Application number
CY20201100405T
Other languages
Greek (el)
English (en)
Inventor
Alessandra Biffi
Bernhard Rudolf Gentner
Luigi Naldini
Original Assignee
Ospedale San Raffaele S.R.L.
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 Ospedale San Raffaele S.R.L. filed Critical Ospedale San Raffaele S.R.L.
Publication of CY1123218T1 publication Critical patent/CY1123218T1/el

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • A61P37/02Immunomodulators
    • A61P37/04Immunostimulants
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/52Cytokines; Lymphokines; Interferons
    • C07K14/555Interferons [IFN]
    • C07K14/56IFN-alpha
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141MicroRNAs, miRNAs
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/51Physical structure in polymeric form, e.g. multimers, concatemers
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special delivery means, e.g. tissue-specific
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2330/00Production
    • C12N2330/50Biochemical production, i.e. in a transformed host cell
    • C12N2330/51Specially adapted vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2799/00Uses of viruses
    • C12N2799/02Uses of viruses as vector
    • C12N2799/021Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/027Uses 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)
  • 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)
CY20201100405T 2009-04-30 2020-05-04 Διαβιβαστης γονιδιου CY1123218T1 (el)

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 (1)

Publication Number Publication Date
CY1123218T1 true CY1123218T1 (el) 2021-10-29

Family

ID=42790671

Family Applications (1)

Application Number Title Priority Date Filing Date
CY20201100405T CY1123218T1 (el) 2009-04-30 2020-05-04 Διαβιβαστης γονιδιου

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)

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PL3360964T3 (pl) 2012-12-06 2020-03-31 Sigma-Aldrich Co. Llc Modyfikacja i regulacja genomu oparta na crispr
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RU2662932C2 (ru) 2013-03-14 2018-07-31 Карибо Биосайенсиз, Инк. Композиции и способы с участием нуклеиновых кислот, нацеленных на нуклеиновые кислоты
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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
US10457940B2 (en) 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
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
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PL2424571T3 (pl) 2009-04-30 2020-10-19 Ospedale San Raffaele S.R.L Wektor genowy

Also Published As

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
EA023938B1 (ru) 2016-07-29
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
CN102596255B (zh) 2017-10-13
KR20120038403A (ko) 2012-04-23
IL215804A0 (en) 2012-01-31
PL2424571T3 (pl) 2020-10-19
JP2016028582A (ja) 2016-03-03
PT2424571T (pt) 2020-05-06
WO2010125471A2 (en) 2010-11-04
US11407996B2 (en) 2022-08-09
SMT202000224T1 (it) 2020-05-08
DK2424571T3 (da) 2020-05-04
SI2424571T1 (sl) 2020-10-30
BRPI1010873A2 (pt) 2016-03-15
HUE049845T2 (hu) 2020-10-28
EP3733214A1 (en) 2020-11-04
HRP20200647T1 (hr) 2020-10-02
JP2012525141A (ja) 2012-10-22
CA2759438A1 (en) 2010-11-04
US9951328B2 (en) 2018-04-24
CA2759438C (en) 2021-03-30
US12577560B2 (en) 2026-03-17
US10287579B2 (en) 2019-05-14
EP2424571A2 (en) 2012-03-07
US20220333103A1 (en) 2022-10-20
JP6144911B2 (ja) 2017-06-07
US20190367911A1 (en) 2019-12-05
AU2010243276A1 (en) 2011-11-17
KR101793615B1 (ko) 2017-11-03

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