AU2017221456B2 - Short hairpin RNA (shRNA734) and use of same to positively select and eliminate genetically modified cells - Google Patents

Short hairpin RNA (shRNA734) and use of same to positively select and eliminate genetically modified cells Download PDF

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Publication number
AU2017221456B2
AU2017221456B2 AU2017221456A AU2017221456A AU2017221456B2 AU 2017221456 B2 AU2017221456 B2 AU 2017221456B2 AU 2017221456 A AU2017221456 A AU 2017221456A AU 2017221456 A AU2017221456 A AU 2017221456A AU 2017221456 B2 AU2017221456 B2 AU 2017221456B2
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cells
polynucleotide
hprt
gene
genetically modified
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AU2017221456A
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AU2017221456A1 (en
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Dong Sung An
Saki Shimizu
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University of California San Diego UCSD
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University of California Berkeley
University of California San Diego UCSD
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    • 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
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
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    • A—HUMAN NECESSITIES
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    • 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
    • A—HUMAN NECESSITIES
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    • 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
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/14—Antivirals for RNA viruses
    • A61P31/18—Antivirals for RNA viruses for HIV
    • A—HUMAN NECESSITIES
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    • 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
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    • 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
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    • 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
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/0081—Purging biological preparations of unwanted cells
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    • 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]
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • C—CHEMISTRY; METALLURGY
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    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted vectors
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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

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  • Health & Medical Sciences (AREA)
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  • Wood Science & Technology (AREA)
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  • Virology (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Cell Biology (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Immunology (AREA)
  • Developmental Biology & Embryology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Hematology (AREA)
  • AIDS & HIV (AREA)
  • Oncology (AREA)
  • Communicable Diseases (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
AU2017221456A 2016-02-19 2017-02-17 Short hairpin RNA (shRNA734) and use of same to positively select and eliminate genetically modified cells Ceased AU2017221456B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662297432P 2016-02-19 2016-02-19
US62/297,432 2016-02-19
PCT/US2017/018483 WO2017143266A1 (en) 2016-02-19 2017-02-17 Short hairpin rna (shrna734) and use of same to positively select and eliminate genetically modified cells

Publications (2)

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AU2017221456A1 AU2017221456A1 (en) 2018-07-19
AU2017221456B2 true AU2017221456B2 (en) 2024-04-04

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AU2017221456A Ceased AU2017221456B2 (en) 2016-02-19 2017-02-17 Short hairpin RNA (shRNA734) and use of same to positively select and eliminate genetically modified cells

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US (2) US11377659B2 (enExample)
EP (1) EP3416660A4 (enExample)
JP (2) JP6975465B2 (enExample)
KR (1) KR20180114008A (enExample)
CN (1) CN108495640B (enExample)
AU (1) AU2017221456B2 (enExample)
HK (1) HK1257668A1 (enExample)
WO (1) WO2017143266A1 (enExample)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES3036762T3 (en) 2017-07-18 2025-09-24 Csl Behring Gene Therapy Inc A modulatable switch for selection of donor modified cells
EP3655534B1 (en) 2017-07-18 2025-07-16 CSL Behring Gene Therapy, Inc. Compositions and methods for treating beta-hemoglobinopathies
JP2022514955A (ja) * 2018-12-23 2022-02-16 シーエスエル・ベーリング・エルエルシー ウィスコット・アルドリッチ症候群の造血幹細胞遺伝子治療
EP3897831A1 (en) * 2018-12-23 2021-10-27 CSL Behring LLC Donor t-cells with kill switch

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2008109837A2 (en) * 2007-03-08 2008-09-12 California Institute Of Technology Method for expression of small antiviral rna molecules with reduced cytotoxicity within a cell

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WO1997043900A1 (en) 1996-05-24 1997-11-27 The President And Fellows Of Harvard College In vivo selection
WO1998019540A1 (en) 1996-11-04 1998-05-14 Saint Jude Children's Research Hospital In vivo selection of primitive hematopoietic cells
US20030032003A1 (en) 2000-02-02 2003-02-13 Schiestl Robert H. In vivo selection
AU2002219828A1 (en) 2000-11-14 2002-05-27 The General Hospital Corporation Blockade of T cell migration into epithelial GVHD target tissues
EP1560931B1 (en) * 2002-11-14 2011-07-27 Dharmacon, Inc. Functional and hyperfunctional sirna
WO2005056761A2 (en) * 2003-12-05 2005-06-23 Massachusetts Institute Of Technology REAGENTS AND METHODS FOR IDENTIFICATION OF RNAi PATHWAY GENES AND CHEMICAL MODULATORS OF RNAi
EP1607482A1 (de) * 2004-06-16 2005-12-21 Gbf-Gesellschaft Für Biotechnologische Forschung Mbh RNAi-basierte Verfahren zur Selektion von transfizierten eukaryontischen Zellen
WO2008076370A2 (en) * 2006-12-18 2008-06-26 The General Hospital Corporation P21 compositions and methods for the treatment of hiv
WO2012061075A2 (en) * 2010-10-25 2012-05-10 The Regents Of The University Of California Hiv resistant and functional hematopoietic stem/progenitor cells and macrophages from induced pluripotent stem cells
GB201301125D0 (en) * 2013-01-22 2013-03-06 Horizon Discovery Ltd Methods for improving the efficency of gene targeting
EP3027755B1 (en) * 2013-08-02 2019-10-09 The Regents of The University of California Engineering antiviral t cell immunity through stem cells and chimeric antigen receptors
KR20180073679A (ko) * 2015-11-04 2018-07-02 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 조혈 줄기 세포에서의 유전자 편집을 위한 방법 및 조성물
EP3655534B1 (en) * 2017-07-18 2025-07-16 CSL Behring Gene Therapy, Inc. Compositions and methods for treating beta-hemoglobinopathies

Patent Citations (1)

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Publication number Priority date Publication date Assignee Title
WO2008109837A2 (en) * 2007-03-08 2008-09-12 California Institute Of Technology Method for expression of small antiviral rna molecules with reduced cytotoxicity within a cell

Also Published As

Publication number Publication date
AU2017221456A1 (en) 2018-07-19
EP3416660A4 (en) 2019-11-06
CN108495640B (zh) 2022-11-04
US20230076635A1 (en) 2023-03-09
US20210310007A1 (en) 2021-10-07
EP3416660A1 (en) 2018-12-26
WO2017143266A1 (en) 2017-08-24
JP7350361B2 (ja) 2023-09-26
KR20180114008A (ko) 2018-10-17
HK1257668A1 (zh) 2019-10-25
CA3012332A1 (en) 2017-08-24
CN108495640A (zh) 2018-09-04
JP6975465B2 (ja) 2021-12-08
JP2022033729A (ja) 2022-03-02
US11377659B2 (en) 2022-07-05
JP2019509034A (ja) 2019-04-04

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