WO2001066150A3 - Highly efficient gene transfer into human repopulating stem cel ls by rd114 pseudotyped retroviral vector particles - Google Patents

Highly efficient gene transfer into human repopulating stem cel ls by rd114 pseudotyped retroviral vector particles Download PDF

Info

Publication number
WO2001066150A3
WO2001066150A3 PCT/US2001/007212 US0107212W WO0166150A3 WO 2001066150 A3 WO2001066150 A3 WO 2001066150A3 US 0107212 W US0107212 W US 0107212W WO 0166150 A3 WO0166150 A3 WO 0166150A3
Authority
WO
WIPO (PCT)
Prior art keywords
vector particles
cells
retroviral vector
human
gene transfer
Prior art date
Application number
PCT/US2001/007212
Other languages
French (fr)
Other versions
WO2001066150A2 (en
WO2001066150A9 (en
Inventor
Patrick F Kelly
Elio F Vanin
Original Assignee
St Jude Childrens Res Hospital
Patrick F Kelly
Elio F Vanin
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 St Jude Childrens Res Hospital, Patrick F Kelly, Elio F Vanin filed Critical St Jude Childrens Res Hospital
Priority to AU2001240078A priority Critical patent/AU2001240078A1/en
Publication of WO2001066150A2 publication Critical patent/WO2001066150A2/en
Publication of WO2001066150A9 publication Critical patent/WO2001066150A9/en
Publication of WO2001066150A3 publication Critical patent/WO2001066150A3/en

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
    • A61K2035/124—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells the cells being hematopoietic, bone marrow derived or blood 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
    • 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
    • C12N2740/13011—Gammaretrovirus, e.g. murine leukeamia virus
    • C12N2740/13041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/13045—Special targeting system for viral 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
    • 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/20—Vectors comprising a special translation-regulating system translation of more than one cistron
    • C12N2840/203—Vectors comprising a special translation-regulating system translation of more than one cistron having an IRES

Abstract

The present invention pertains to a method for efficiently introducing exogenous genes into stem cells, particularly human stem cells. The method optionally includes the steps of inducing the proliferation of target cells by pre-stimulation with cytokines and/or growth factors, followed by incubating these cells with RD114-pseudotyped vector particles. In a specific embodiment, the vector particles are retronectin-immobilzed or ultracentrifugation-concentrated retroviral vector particles pseudotyped with the feline endogenous retrovirus (RD114) envelope protein. The present invention further discloses a method for somatic gene therapy, which can be used for various therapeutic applications and involves introducing a gene of interest contained within the retroviral genome into human repopulating stem cells followed by introducing these cells into a human host. Finally, the present invention discloses a method for monitoring the efficiency of the stem cell-mediated gene transfer based on detecting the presence of the genes (or the expression products) of the retroviral vector in various stem cell-derived lineages of the host.
PCT/US2001/007212 2000-03-07 2001-03-07 Highly efficient gene transfer into human repopulating stem cel ls by rd114 pseudotyped retroviral vector particles WO2001066150A2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2001240078A AU2001240078A1 (en) 2000-03-07 2001-03-07 Highly efficient gene transfer into human repopulating stem cells by rd114 pseudotyped retroviral vector particles

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US18753400P 2000-03-07 2000-03-07
US60/187,534 2000-03-07

Publications (3)

Publication Number Publication Date
WO2001066150A2 WO2001066150A2 (en) 2001-09-13
WO2001066150A9 WO2001066150A9 (en) 2002-02-28
WO2001066150A3 true WO2001066150A3 (en) 2002-05-02

Family

ID=22689373

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2001/007212 WO2001066150A2 (en) 2000-03-07 2001-03-07 Highly efficient gene transfer into human repopulating stem cel ls by rd114 pseudotyped retroviral vector particles

Country Status (2)

Country Link
AU (1) AU2001240078A1 (en)
WO (1) WO2001066150A2 (en)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB0400443D0 (en) 2004-01-09 2004-02-11 Oxford Biomedica Ltd Cascade
JP2005503162A (en) * 2001-09-21 2005-02-03 オックスフォード バイオメディカ（ユーケイ）リミテッド vector
EP2019144A1 (en) * 2007-07-23 2009-01-28 Institut National De La Sante Et De La Recherche Medicale (Inserm) Vector particles for targeting CD34+ cells
KR20160075676A (en) * 2013-10-24 2016-06-29 오스페달레 산 라파엘 에스.알.엘. Method

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1996004934A1 (en) * 1994-08-17 1996-02-22 Genetic Therapy Inc. Retroviral vectors produced by producer cell lines resistant to lysis by human serum
WO1999015684A2 (en) * 1997-09-23 1999-04-01 Oxford Biomedica (Uk) Limited Expression of genes in hematopoietic stem cells in hischaemic conditions
WO1999032646A1 (en) * 1997-12-22 1999-07-01 Oxford Biomedica (Uk) Limited Equine infectious anaemia virus (eiav) based

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1996004934A1 (en) * 1994-08-17 1996-02-22 Genetic Therapy Inc. Retroviral vectors produced by producer cell lines resistant to lysis by human serum
WO1999015684A2 (en) * 1997-09-23 1999-04-01 Oxford Biomedica (Uk) Limited Expression of genes in hematopoietic stem cells in hischaemic conditions
WO1999032646A1 (en) * 1997-12-22 1999-07-01 Oxford Biomedica (Uk) Limited Equine infectious anaemia virus (eiav) based

Non-Patent Citations (9)

* Cited by examiner, † Cited by third party
Title
COSSET F-L ET AL: "HIGH-TITER PACKAGING CELLS PRODUCING RECOMBINANT RETROVIRUSES RESISTANT TO HUMAN SERUM", JOURNAL OF VIROLOGY, THE AMERICAN SOCIETY FOR MICROBIOLOGY, US, vol. 69, no. 12, 1 December 1995 (1995-12-01), pages 7430 - 7436, XP000569527, ISSN: 0022-538X *
HANAWA H ET AL: "Improved transduction of human primitive hematopoietic cells with a lentiviral vector pseudotyped with the envelope protein of endogenous feline leukemia virus (RD114)", BLOOD, vol. 96, no. 11 Part.1, 16 November 2000 (2000-11-16), pages 524a, XP002190048 *
HANENBERG H ET AL: "OPTIMIZATION OF FIBRONECTIN-ASSISTED RETROVIRAL GENE TRANSFER INTO HUMAN CD34+ HEMATOPOIETIC CELLS", HUMAN GENE THERAPY, XX, XX, vol. 8, no. 18, 10 December 1997 (1997-12-10), pages 2193 - 2206, XP000867308, ISSN: 1043-0342 *
HENNEMANN B ET AL: "OPTIMIZATION OF RETROVIRAL-MEDIATED GENE TRANSFER TO HUMAN NOD/SCID MOUSE REPOPULATING CORD BLOOD CELLS THROUGH A SYSTEMATIC ANALYSIS OF PROTOCOL VARIABLES", EXPERIMENTAL HEMATOLOGY, NEW YORK, NY, US, vol. 27, no. 5, May 1999 (1999-05-01), pages 817 - 825, XP000867307, ISSN: 0301-472X *
KELLY P ET AL: "Efficient transduction of CD34+ and CD34+, CD38- human haematopoietic cells with SCID repopulating cells (SRC) potential with an oncoretroviral vector pseudotyped with a feline endogenous virus (RD114) envelope protein", BLOOD, vol. 94, no. 10 Part 1 Supl.1, 15 November 1999 (1999-11-15), pages 611a; Abs. 2718, XP002190046 *
KELLY P ET AL: "Highly efficient gene transfer into cord blood nonobese diabetic/severe combined immunodeficiency repopulating cells by oncoretroviral vector particles pseudotyped with the feline endogenous retrovirus (RD114) envelope protein", BLOOD, vol. 96, no. 4, 15 August 2000 (2000-08-15), pages 1206 - 1214, XP002190047 *
KIEM H ET AL: "Improved gene transfer into baboon marrow repopulating cells using recombinant human fibronectin fragment CH-296 in combination with interleukin-6, stem cell factor, FLT-3 ligand, and megakaryocyte growth and development factor", BLOOD, vol. 92, no. 6, 15 September 1998 (1998-09-15), pages 1878 - 1886, XP002190050 *
MIYOSHI H ET AL: "Transduction of human CD34+ cells that mediate long-term engraftment of NOD/SCID mice by HIV vectors", SCIENCE, vol. 283, no. 5402, 29 January 1999 (1999-01-29), pages 682 - 686, XP002190051 *
PORTER C ET AL: "Comparison of efficiency of infection of human gene therapy target cells via four different retroviral receptors", HUM GENE THER, vol. 7, no. 8, 20 May 1996 (1996-05-20), pages 913 - 919, XP001053542 *

Also Published As

Publication number Publication date
AU2001240078A1 (en) 2001-09-17
WO2001066150A2 (en) 2001-09-13
WO2001066150A9 (en) 2002-02-28

Similar Documents

Publication Publication Date Title
CN106480036B (en) A kind of DNA fragmentation and its application with promoter function
EP1291419A4 (en) Pseudo-type retrovirus vector containing membrane protein having hemagglutinin activity
ATE198767T1 (en) DUMBBELL SHAPED EXPRESSION CONSTRUCTS FOR GENE THERAPY
EP1196426A4 (en) Cytoplasmic transfer to de-differentiate recipient cells
EP3704237A1 (en) Novel crispr-associated transposon systems and components
BR8200191A (en) LIVING CELL RENIN PREPRORRENIN PRORRENIN PROCESS FOR OBTAINING EXPRESSION OF PREPORRENIN PRORRENIN AND RENIN PREORGENIN AND RENIN AND COLI GENERATING MATERIAL DNA RECOMBINATOR SACCHAROMYCES CEREVISIAE POLIPETIDIME PROCESS AND A POLYPETHYDICAL PROCESS FOR A PROCESS
ES2255281T3 (en) OVEREXPRESSION OF FITASA GENES IN SISTMAS DE LEVADURA.
BR0013549A (en) Phytase enzymes, nucleic acids encoding phytase enzymes and vectors and host cells incorporating them
EP0752874A4 (en) Enhanced virus-mediated dna transfer
WO2003093469A3 (en) Methods for delivering nucleic acid molecules into cells and assessment thereof
HUP9900868A2 (en) Chromosomal expression of heterologous genes in bacterial cells
WO1997005255A3 (en) Helper viruses for preparing recombinant viral vectors
AU4777900A (en) Rnp originating in paramyxovirus
AUPN477695A0 (en) Gene therapy
JP2008086326A (en) Gene manipulation and expression using genomic element
WO2005079367A3 (en) Schizochytrium fatty acid synthase (fas) and products and methods related thereto
CN109321548A (en) A kind of Cas9 albumen, CRISPR/Cas9 system, the method and application of mushroom gene editing
WO2001066150A3 (en) Highly efficient gene transfer into human repopulating stem cel ls by rd114 pseudotyped retroviral vector particles
CA2037677A1 (en) Process for production of exogenous gene or its product in plant cells
CN104152414A (en) Gene modifying method for reserved locus of cell genome
WO2005007803A3 (en) Selection free growth of host cells containing multiple integrating vectors
CN105274089B (en) A kind of construction method for the REV viral infectivities clone for expressing green fluorescence cyst membrane fusion protein
CN206204267U (en) Kit of the one kind for mammalian cell " gene editing " vector construction
WO2000049166A3 (en) Production of helper dependent adenovirus vectors based on use of endonucleases
WO2001025398A3 (en) Process for inducing functional tolerance to gene transfer products

Legal Events

Date Code Title Description
AK Designated states

Kind code of ref document: A2

Designated state(s): AE AG AL AM AT AU AZ BA BB BG BR BY BZ CA CH CN CO CR CU CZ DE DK DM EE ES FI GB GD GE HR HU ID IL IN IS JP KE KG KP KR KZ LC LK LR LS LT LU LV MA MD MG MK MN MW MX MZ NO NZ PL PT RO RU SD SE SG SI SK SL TJ TM TR TT TZ UA UG US UZ VN YU ZA ZW

AL Designated countries for regional patents

Kind code of ref document: A2

Designated state(s): GH GM KE LS MW MZ SD SL SZ TZ UG ZW AM AZ BY KG KZ MD RU TJ TM AT BE CH CY DE DK ES FI FR GB GR IE IT LU MC NL PT SE TR BF BJ CF CG CI CM GA GN GW ML MR NE SN TD TG

121 Ep: the epo has been informed by wipo that ep was designated in this application
DFPE Request for preliminary examination filed prior to expiration of 19th month from priority date (pct application filed before 20040101)
AK Designated states

Kind code of ref document: C2

Designated state(s): AE AG AL AM AT AU AZ BA BB BG BR BY BZ CA CH CN CO CR CU CZ DE DK DM EE ES FI GB GD GE HR HU ID IL IN IS JP KE KG KP KR KZ LC LK LR LS LT LU LV MA MD MG MK MN MW MX MZ NO NZ PL PT RO RU SD SE SG SI SK SL TJ TM TR TT TZ UA UG US UZ VN YU ZA ZW

AL Designated countries for regional patents

Kind code of ref document: C2

Designated state(s): GH GM KE LS MW MZ SD SL SZ TZ UG ZW AM AZ BY KG KZ MD RU TJ TM AT BE CH CY DE DK ES FI FR GB GR IE IT LU MC NL PT SE TR BF BJ CF CG CI CM GA GN GW ML MR NE SN TD TG

COP Corrected version of pamphlet

Free format text: PAGE 34, DESCRIPTION, REPLACED BY A NEW PAGE 34; AFTER RECTIFICATION OF OBVIOUS ERRORS AS AUTHORIZED BY THE INTERNATIONAL SEARCHING AUTHORITY

REG Reference to national code

Ref country code: DE

Ref legal event code: 8642

122 Ep: pct application non-entry in european phase
NENP Non-entry into the national phase

Ref country code: JP