PL2424571T3 - Wektor genowy - Google Patents

Wektor genowy

Info

Publication number
PL2424571T3
PL2424571T3 PL10723305T PL10723305T PL2424571T3 PL 2424571 T3 PL2424571 T3 PL 2424571T3 PL 10723305 T PL10723305 T PL 10723305T PL 10723305 T PL10723305 T PL 10723305T PL 2424571 T3 PL2424571 T3 PL 2424571T3
Authority
PL
Poland
Prior art keywords
gene vector
gene
vector
Prior art date
Application number
PL10723305T
Other languages
English (en)
Inventor
Alessandra Biffi
Bernhard Rudolf Gentner
Luigi Naldini
Original Assignee
Ospedale San Raffaele S.R.L
Fondazione Telethon
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, Fondazione Telethon filed Critical Ospedale San Raffaele S.R.L
Publication of PL2424571T3 publication Critical patent/PL2424571T3/pl

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
    • C12N15/63Introduction 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
    • C12N15/11DNA 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
    • 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 N.A.
    • 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)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (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)
PL10723305T 2009-04-30 2010-04-30 Wektor genowy PL2424571T3 (pl)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US17412409P 2009-04-30 2009-04-30
EP10723305.8A EP2424571B1 (en) 2009-04-30 2010-04-30 Gene vector
PCT/IB2010/001166 WO2010125471A2 (en) 2009-04-30 2010-04-30 Gene vector

Publications (1)

Publication Number Publication Date
PL2424571T3 true PL2424571T3 (pl) 2020-10-19

Family

ID=42790671

Family Applications (1)

Application Number Title Priority Date Filing Date
PL10723305T PL2424571T3 (pl) 2009-04-30 2010-04-30 Wektor genowy

Country Status (22)

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

Families Citing this family (34)

* 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
EP2569436B8 (en) 2010-05-14 2019-10-30 Oregon Health & Science University Recombinant hcmv and rhcmv vectors and uses thereof
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
ES2960803T3 (es) 2012-05-25 2024-03-06 Univ California Métodos y composiciones para la modificación de ADN diana dirigida por RNA y para la modulación de la transcripción dirigida por RNA
EP2872154B1 (en) 2012-07-11 2017-05-31 Sangamo BioSciences, Inc. Methods and compositions for delivery of biologics
EP3617309A3 (en) 2012-12-06 2020-05-06 Sigma Aldrich Co. LLC Crispr-based genome modification and regulation
HRP20231183T1 (hr) 2013-02-15 2024-01-05 Bioverativ Therapeutics Inc. Optimizirani gen faktora viii
AU2014235794A1 (en) 2013-03-14 2015-10-22 Caribou Biosciences, Inc. Compositions and methods of nucleic acid-targeting nucleic acids
AU2014338555B2 (en) 2013-10-24 2019-10-10 Fondazione Telethon Method
EP3160478A4 (en) 2014-06-30 2018-05-16 Bioverativ Therapeutics Inc. Optimized factor ix gene
WO2016171235A1 (ja) * 2015-04-22 2016-10-27 国立大学法人京都大学 組織細胞の選別方法
WO2016201507A1 (en) * 2015-06-15 2016-12-22 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
SI3411478T1 (sl) 2016-02-01 2022-10-28 Bioverativ Therapeutics Inc. Optimirani geni dejavnika VIII
US10457940B2 (en) 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
SG11201903340TA (en) * 2016-10-18 2019-05-30 Univ Oregon Health & Science 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
CA3060145A1 (en) * 2017-04-21 2018-10-25 Ospedale San Raffaele S.R.L. Cytokine gene therapy for treating or preventing cancer
IT201700086403A1 (it) * 2017-07-27 2019-01-27 Univ Del Piemonte Orientale Endothelial-specific promoter sequences and uses thereof
US20210163986A1 (en) 2017-08-09 2021-06-03 Bioverativ Therapeutics Inc. Nucleic acid molecules and uses thereof
SG11202007114VA (en) 2018-02-01 2020-08-28 Bioverativ Therapeutics Inc Use of lentiviral vectors expressing factor viii
CA3108799A1 (en) 2018-08-09 2020-02-13 Bioverativ Therapeutics Inc. Nucleic acid molecules and uses thereof for non-viral gene therapy
JP2022514465A (ja) 2018-12-06 2022-02-14 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
WO2021067389A1 (en) 2019-09-30 2021-04-08 Bioverativ Therapeutics Inc. Lentiviral vector formulations
WO2021097109A1 (en) * 2019-11-12 2021-05-20 The Regents Of The University Of California Lentiviral vectors in hematopoietic stem cells to treat x-linked chronic granulomatous disease
TW202208619A (zh) 2020-06-24 2022-03-01 美商百歐維拉提夫治療公司 用於純化病毒載體之方法
US20220090130A1 (en) 2020-08-23 2022-03-24 Bioverativ Therapeutics Inc. MODIFIED BACULOVIRUS SYSTEM FOR IMPROVED PRODUCTION OF CLOSED-ENDED DNA (ceDNA)
IL310997A (en) 2021-08-23 2024-04-01 Bioverativ Therapeutics Inc Factor VIII gene optimization
KR20240049821A (ko) 2021-08-23 2024-04-17 바이오버라티브 테라퓨틱스 인크. 바큘로바이러스 발현 시스템
IL311725A (en) 2021-09-30 2024-05-01 Bioverativ Therapeutics Inc Nucleic acids encoding factor VIII polypeptides with reduced immunogenicity
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
AU730464B2 (en) 1996-08-07 2001-03-08 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
CA2915676C (en) * 2001-10-02 2017-06-06 Institut Clayton De La Recherche Restricted expression lentiviral vectors
EP1771563A2 (en) 2004-05-28 2007-04-11 Ambion, 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
EP2002003B1 (en) * 2005-05-27 2015-12-30 Ospedale San Raffaele S.r.l. Gene vector comprising mi-rna
EP2377559A1 (en) * 2005-06-03 2011-10-19 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
EP2487257B1 (en) 2006-01-05 2015-07-01 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
ATE534738T1 (de) 2006-12-08 2011-12-15 Asuragen Inc Mir-126-regulierte gene und pfade als ziele für therapeutische interventionen
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
PL2424571T3 (pl) 2009-04-30 2020-10-19 Ospedale San Raffaele S.R.L Wektor genowy

Also Published As

Publication number Publication date
US20120128643A1 (en) 2012-05-24
IL215804A0 (en) 2012-01-31
US20220333103A1 (en) 2022-10-20
KR20120038403A (ko) 2012-04-23
JP2016028582A (ja) 2016-03-03
US11407996B2 (en) 2022-08-09
LT2424571T (lt) 2020-08-10
BRPI1010873B1 (pt) 2020-04-14
JP2012525141A (ja) 2012-10-22
SG175839A1 (en) 2011-12-29
CA2759438C (en) 2021-03-30
EA201171335A1 (ru) 2012-05-30
AU2010243276A1 (en) 2011-11-17
CA2759438A1 (en) 2010-11-04
US9951328B2 (en) 2018-04-24
JP6144911B2 (ja) 2017-06-07
HUE049845T2 (hu) 2020-10-28
EA023938B1 (ru) 2016-07-29
IL215804A (en) 2016-10-31
AU2010243276B2 (en) 2016-09-15
CY1123218T1 (el) 2021-10-29
SI2424571T1 (sl) 2020-10-30
US20190367911A1 (en) 2019-12-05
KR101793615B1 (ko) 2017-11-03
JP6370278B2 (ja) 2018-08-08
HRP20200647T1 (hr) 2020-10-02
ES2786039T3 (es) 2020-10-08
EP2424571B1 (en) 2020-04-08
DK2424571T3 (da) 2020-05-04
CN102596255A (zh) 2012-07-18
US10287579B2 (en) 2019-05-14
WO2010125471A3 (en) 2010-12-29
WO2010125471A2 (en) 2010-11-04
SG10201610827RA (en) 2017-02-27
EP3733214A1 (en) 2020-11-04
PT2424571T (pt) 2020-05-06
BRPI1010873A2 (pt) 2016-03-15
CN102596255B (zh) 2017-10-13
US20180044670A1 (en) 2018-02-15
EP2424571A2 (en) 2012-03-07
MX2011011508A (es) 2012-02-13

Similar Documents

Publication Publication Date Title
IL215804A0 (en) Gene vector
ZA201202407B (en) Photobioreactor
GB201103481D0 (en) No details
GB201016096D0 (en) No details
EP2380416A4 (en) CARRYING CASE
EP2459716A4 (en) ADENOVIRAL BASED VECTORS
ZA201206766B (en) Lance
GB201016097D0 (en) No details
GB201118187D0 (en) Photobioreactor
GB201007384D0 (en) Enzymes
PL2464939T3 (pl) Udoskonalony nośnik przedmiotu obrabianego
GB0810912D0 (en) Vector
EP2516615A4 (en) IMPROVED BIOREACTORS
GB0914596D0 (en) Improved hatchcover
GB201004475D0 (en) Gene silencing
PT2274422T (pt) Vetor de expressão
PL2664278T3 (pl) Lancet
GB0903658D0 (en) Antimicobial coatings
GB0908435D0 (en) Processes
GB2471314B (en) Killswitch
PL2393603T3 (pl) Lanca
GB0810154D0 (en) Expression vector
GB0910833D0 (en) Avian genes
GB201105987D0 (en) Enzymes
IL208602A0 (en) Mezuzah case