KR20180114008A - 짧은 헤어핀 rna(shrna734) 및 유전자 변형된 세포를 양성으로 선택하고 제거하기 위한 이의 용도 - Google Patents
짧은 헤어핀 rna(shrna734) 및 유전자 변형된 세포를 양성으로 선택하고 제거하기 위한 이의 용도 Download PDFInfo
- Publication number
- KR20180114008A KR20180114008A KR1020187021571A KR20187021571A KR20180114008A KR 20180114008 A KR20180114008 A KR 20180114008A KR 1020187021571 A KR1020187021571 A KR 1020187021571A KR 20187021571 A KR20187021571 A KR 20187021571A KR 20180114008 A KR20180114008 A KR 20180114008A
- Authority
- KR
- South Korea
- Prior art keywords
- cells
- genetically modified
- polynucleotide
- gene
- cell
- 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.)
- Ceased
Links
Images
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
-
- 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
-
- 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
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- 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
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- 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
-
- 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
- C12N15/11—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
-
- 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
- C12N15/11—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
-
- 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
-
- 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
- C12N5/0081—Purging biological preparations of unwanted 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
- 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]
-
- 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
- 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
- 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
- C12N2330/00—Production
- C12N2330/50—Biochemical production, i.e. in a transformed host cell
- C12N2330/51—Specially adapted 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
- 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
- C12Y—ENZYMES
- C12Y204/00—Glycosyltransferases (2.4)
- C12Y204/02—Pentosyltransferases (2.4.2)
- C12Y204/02008—Hypoxanthine phosphoribosyltransferase (2.4.2.8)
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Virology (AREA)
- Medicinal Chemistry (AREA)
- 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)
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 (1)
| Publication Number | Publication Date |
|---|---|
| KR20180114008A true KR20180114008A (ko) | 2018-10-17 |
Family
ID=59625420
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020187021571A Ceased KR20180114008A (ko) | 2016-02-19 | 2017-02-17 | 짧은 헤어핀 rna(shrna734) 및 유전자 변형된 세포를 양성으로 선택하고 제거하기 위한 이의 용도 |
Country Status (8)
| Country | Link |
|---|---|
| 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)
| 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 |
Family Cites Families (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| US7737124B2 (en) * | 2001-09-13 | 2010-06-15 | California Institute Of Technology | Method for expression of small antiviral RNA molecules with reduced cytotoxicity within a cell |
| 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 |
-
2017
- 2017-02-17 CN CN201780007698.1A patent/CN108495640B/zh active Active
- 2017-02-17 AU AU2017221456A patent/AU2017221456B2/en not_active Ceased
- 2017-02-17 KR KR1020187021571A patent/KR20180114008A/ko not_active Ceased
- 2017-02-17 JP JP2018543719A patent/JP6975465B2/ja active Active
- 2017-02-17 EP EP17753977.2A patent/EP3416660A4/en active Pending
- 2017-02-17 US US15/999,854 patent/US11377659B2/en active Active
- 2017-02-17 HK HK19100028.5A patent/HK1257668A1/zh unknown
- 2017-02-17 WO PCT/US2017/018483 patent/WO2017143266A1/en not_active Ceased
-
2021
- 2021-10-29 JP JP2021177252A patent/JP7350361B2/ja active Active
-
2022
- 2022-06-03 US US17/805,344 patent/US20230076635A1/en active Pending
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 |
| 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 |
| AU2017221456B2 (en) | 2024-04-04 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP7350361B2 (ja) | 遺伝子改変細胞をポジティブに選択して、排除するための短ヘアピンrna(shrna734)およびその使用 | |
| US11679131B2 (en) | Genetically engineered T cells with regnase-1 and/or TGFBRII disruption have improved functionality and persistence | |
| US20200299661A1 (en) | Cpf1-related methods and compositions for gene editing | |
| US20240167059A1 (en) | Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites | |
| JP2013165733A (ja) | H19をダウンレギュレートする核酸薬剤、及びそれを使用する方法 | |
| JP2024519524A (ja) | 疾患の処置のために有用なレンチウイルスベクター | |
| JP2018515136A (ja) | B型肝炎ウイルス(hbv)感染を治療するための試薬及びその使用 | |
| US20240368582A1 (en) | Modification of epor-encoding nucleic acids | |
| CA3216023A1 (en) | Adenoviral gene therapy vectors | |
| US20200263206A1 (en) | Targeted integration systems and methods for the treatment of hemoglobinopathies | |
| JP2022514954A (ja) | キルスイッチを有するドナーt細胞 | |
| AU2021256428A1 (en) | Integration of large adenovirus payloads | |
| CA3012332C (en) | Short hairpin rna (shrna734) and use of same to positively select and eliminate genetically modified cells | |
| CN119585419A (zh) | 工程化t细胞 | |
| US20240050435A1 (en) | Methods for treating cancer | |
| US20240124840A1 (en) | Genetically modified cells and uses thereof for prevention of acute graft-versus-host disease | |
| WO2024059821A2 (en) | Car t cell compositions for treatment of cancer | |
| WO2024249346A9 (en) | Crispr-related methods and compositions targeting cd70 expression | |
| WO2024249342A1 (en) | Crispr-related methods and compositions targeting ptpn2 expression | |
| WO2024249348A2 (en) | Crispr-related methods and compositions targeting fl1-1 expression | |
| Cradick et al. | 407. Zinc-Finger Nucleases Targeting Hepatitis B Virus Genomic DNA |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| PA0105 | International application |
Patent event date: 20180725 Patent event code: PA01051R01D Comment text: International Patent Application |
|
| PG1501 | Laying open of application | ||
| PA0201 | Request for examination |
Patent event code: PA02012R01D Patent event date: 20220217 Comment text: Request for Examination of Application |
|
| E902 | Notification of reason for refusal | ||
| PE0902 | Notice of grounds for rejection |
Comment text: Notification of reason for refusal Patent event date: 20240122 Patent event code: PE09021S01D |
|
| E601 | Decision to refuse application | ||
| PE0601 | Decision on rejection of patent |
Patent event date: 20240726 Comment text: Decision to Refuse Application Patent event code: PE06012S01D |