CN110769860B - 基因疗法 - Google Patents

基因疗法 Download PDF

Info

Publication number
CN110769860B
CN110769860B CN201880038804.7A CN201880038804A CN110769860B CN 110769860 B CN110769860 B CN 110769860B CN 201880038804 A CN201880038804 A CN 201880038804A CN 110769860 B CN110769860 B CN 110769860B
Authority
CN
China
Prior art keywords
cells
cell
vector
csh
transduction
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
CN201880038804.7A
Other languages
English (en)
Chinese (zh)
Other versions
CN110769860A (zh
Inventor
A.C.卡加斯特-鲁德尼茨基
C.佩特里洛
B.R.金特纳
L.纳尔迪尼
P.吉诺维斯
G.希罗里
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.)
IRCCS Ospedale San Raffaele
Original Assignee
IRCCS Ospedale San Raffaele
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 IRCCS Ospedale San Raffaele filed Critical IRCCS Ospedale San Raffaele
Publication of CN110769860A publication Critical patent/CN110769860A/zh
Application granted granted Critical
Publication of CN110769860B publication Critical patent/CN110769860B/zh
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

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
    • 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
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem 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
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • 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
    • A61K48/0083—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the administration regime
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K7/00—Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
    • C07K7/64—Cyclic peptides containing only normal peptide links
    • C07K7/645—Cyclosporins; Related peptides
    • 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
    • C12N15/67—General methods for enhancing the expression
    • C12N15/69—Increasing the copy number of the vector
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0647—Haematopoietic stem cells; Uncommitted or multipotent progenitors
    • 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
    • C12N2510/00—Genetically modified cells
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/13011—Gammaretrovirus, e.g. murine leukeamia virus
    • C12N2740/13041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/13043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/60—Vectors comprising as targeting moiety peptide derived from defined protein from viruses
    • C12N2810/6045—RNA rev transcr viruses
    • C12N2810/6054—Retroviridae
    • 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
    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/60—Vectors comprising as targeting moiety peptide derived from defined protein from viruses
    • C12N2810/6072—Vectors comprising as targeting moiety peptide derived from defined protein from viruses negative strand RNA viruses
    • C12N2810/6081—Vectors comprising as targeting moiety peptide derived from defined protein from viruses negative strand RNA viruses rhabdoviridae, e.g. VSV

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Hematology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Cell Biology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
CN201880038804.7A 2017-04-21 2018-04-20 基因疗法 Active CN110769860B (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GB1706394.2 2017-04-21
GBGB1706394.2A GB201706394D0 (en) 2017-04-21 2017-04-21 Gene Therapy
PCT/EP2018/060237 WO2018193118A1 (en) 2017-04-21 2018-04-20 Gene therapy

Publications (2)

Publication Number Publication Date
CN110769860A CN110769860A (zh) 2020-02-07
CN110769860B true CN110769860B (zh) 2024-03-08

Family

ID=58795636

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201880038804.7A Active CN110769860B (zh) 2017-04-21 2018-04-20 基因疗法

Country Status (18)

Country Link
US (2) US11964027B2 (enExample)
EP (2) EP3612237B1 (enExample)
JP (2) JP7443063B2 (enExample)
CN (1) CN110769860B (enExample)
AU (1) AU2018253958B2 (enExample)
CA (1) CA3060128A1 (enExample)
DK (1) DK3612237T3 (enExample)
ES (1) ES2900560T3 (enExample)
GB (1) GB201706394D0 (enExample)
HR (1) HRP20211803T1 (enExample)
HU (1) HUE056570T2 (enExample)
IL (1) IL270032B2 (enExample)
LT (1) LT3612237T (enExample)
PL (1) PL3612237T3 (enExample)
PT (1) PT3612237T (enExample)
RS (1) RS62619B1 (enExample)
SI (1) SI3612237T1 (enExample)
WO (1) WO2018193118A1 (enExample)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2850484C (en) * 2011-09-30 2021-01-26 Bluebird Bio, Inc. Compounds for improved viral transduction
WO2023077107A1 (en) * 2021-10-29 2023-05-04 Sana Biotechnology, Inc. Methods and reagents for amplifying viral vector nucleic acid products
GB202208550D0 (en) * 2022-06-10 2022-07-27 Ospedale San Raffaele Srl Cyclosporin h and uses thereof
GB202209098D0 (en) * 2022-06-21 2022-08-10 Ucl Business Ltd Cyclosporine analogues
JP2025530652A (ja) 2022-08-11 2025-09-17 フォンダジオン テレソン イーティーエス 遺伝子治療
EP4602153A1 (en) 2022-10-11 2025-08-20 Fondazione Telethon ETS 3d cell culture methods
IT202300011790A1 (it) 2023-06-08 2024-12-08 Fond Telethon Ets Protocolli di manipolazione genica in cellule immunitarie
IT202300022191A1 (it) 2023-10-24 2025-04-24 Fond Telethon Ets Mezzi e metodi per la modifica genetica di uba1
EP4640830A1 (en) 2024-04-23 2025-10-29 Fondazione Telethon ETS Means and methods for safe and efficient gene editing in cells

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2004098531A2 (en) * 2003-05-05 2004-11-18 Virxsys Corporation Increased transduction using abc transporter substrates and/or inhibitors
WO2015162594A2 (en) * 2014-04-25 2015-10-29 Ospedale San Raffaele S.R.L. Gene therapy

Family Cites Families (11)

* 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
CZ297278B6 (cs) 1996-08-07 2006-10-11 Darwin Discovery Limited Deriváty hydroxamové a karboxylové kyseliny a jejich pouzití
US6126939A (en) 1996-09-03 2000-10-03 Yeda Research And Development Co. Ltd. Anti-inflammatory dipeptide and pharmaceutical composition thereof
PT904392E (pt) 1996-10-17 2001-06-29 Oxford Biomedica Ltd Vectores retrovirais
ATE368122T1 (de) 1999-04-29 2007-08-15 Cell Genesys Inc Verfahren und mittel für die herstellung von sicheren rekombinanten lentiviralen vektoren mit hohem titer
ATE527347T1 (de) 2001-08-02 2011-10-15 Inst Clayton De La Rech Verfahren und zusammensetzungen im zusammenhang mit verbesserten lentivirusvektor- produktionssystemen
JP2006513153A (ja) 2002-10-09 2006-04-20 ロランティス リミテッド 免疫機能の調節
US9669049B2 (en) 2010-10-31 2017-06-06 Tocagen Inc. Compositions comprising gamma retrovirus vectors and methods of treating proliferative disorders
WO2014109728A1 (en) 2013-01-11 2014-07-17 The Scripps Research Institute Methods and compositions for enchancing transduction efficiency of retroviral vectors
ES2747951T3 (es) 2013-10-24 2020-03-12 Ospedale San Raffaele Srl Método
CA2986310A1 (en) 2015-05-11 2016-11-17 Editas Medicine, Inc. Optimized crispr/cas9 systems and methods for gene editing in stem cells

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2004098531A2 (en) * 2003-05-05 2004-11-18 Virxsys Corporation Increased transduction using abc transporter substrates and/or inhibitors
WO2015162594A2 (en) * 2014-04-25 2015-10-29 Ospedale San Raffaele S.R.L. Gene therapy

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
SIRT1在内皮细胞中抑制PMA和ionomycin诱导的ICAM-1的表达;贾玉艳等;《中国科学:生命科学》;20130220(第02期);第14-20页 *

Also Published As

Publication number Publication date
WO2018193118A1 (en) 2018-10-25
US12485187B2 (en) 2025-12-02
CA3060128A1 (en) 2018-10-25
US20210121579A1 (en) 2021-04-29
JP2024045128A (ja) 2024-04-02
CN110769860A (zh) 2020-02-07
JP2020517253A (ja) 2020-06-18
EP3612237A1 (en) 2020-02-26
RS62619B1 (sr) 2021-12-31
HRP20211803T1 (hr) 2022-03-04
HUE056570T2 (hu) 2022-02-28
LT3612237T (lt) 2022-01-25
AU2018253958B2 (en) 2024-03-07
IL270032A (enExample) 2019-12-31
DK3612237T3 (da) 2021-12-06
EP3612237B1 (en) 2021-09-01
ES2900560T3 (es) 2022-03-17
SI3612237T1 (sl) 2022-02-28
EP3973996A1 (en) 2022-03-30
GB201706394D0 (en) 2017-06-07
IL270032B1 (en) 2024-11-01
IL270032B2 (en) 2025-03-01
US20240350661A1 (en) 2024-10-24
PL3612237T3 (pl) 2022-02-14
US11964027B2 (en) 2024-04-23
JP7443063B2 (ja) 2024-03-05
AU2018253958A1 (en) 2019-11-21
PT3612237T (pt) 2021-12-02

Similar Documents

Publication Publication Date Title
US12485187B2 (en) Method for improving retroviral transduction and gene editing in hematopoietic stem cells using clyclosporin H and UM171
JP6691048B2 (ja) 方法
US20200237962A1 (en) Gene Therapy
US20240408145A1 (en) Gene Therapy
US12558435B2 (en) Transient inhibition of p53 in gene therapy
EP2943203A1 (en) Methods and compositions for enchancing transduction efficiency of retroviral vectors
US20240218330A1 (en) Compositions for improving the transduction of cells by viral vectors
JP7320950B2 (ja) 遺伝子治療
Li et al. Enhanced genetic modification of adult growth factor mobilized peripheral blood hematopoietic stem and progenitor cells with rapamycin

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination
GR01 Patent grant
GR01 Patent grant