JP2019509034A5 - - Google Patents
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- Publication number
- JP2019509034A5 JP2019509034A5 JP2018543719A JP2018543719A JP2019509034A5 JP 2019509034 A5 JP2019509034 A5 JP 2019509034A5 JP 2018543719 A JP2018543719 A JP 2018543719A JP 2018543719 A JP2018543719 A JP 2018543719A JP 2019509034 A5 JP2019509034 A5 JP 2019509034A5
- Authority
- JP
- Japan
- Prior art keywords
- cells
- polynucleotide
- cell
- genetically modified
- seq
- 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.)
- Granted
Links
- 210000004027 cell Anatomy 0.000 claims 31
- 108091033319 polynucleotide Proteins 0.000 claims 19
- 102000040430 polynucleotide Human genes 0.000 claims 19
- 239000002157 polynucleotide Substances 0.000 claims 19
- 238000000034 method Methods 0.000 claims 15
- 108090000623 proteins and genes Proteins 0.000 claims 8
- 230000000340 anti-metabolite Effects 0.000 claims 5
- 229940100197 antimetabolite Drugs 0.000 claims 5
- 239000002256 antimetabolite Substances 0.000 claims 5
- UYTPUPDQBNUYGX-UHFFFAOYSA-N guanine Chemical class O=C1NC(N)=NC2=C1N=CN2 UYTPUPDQBNUYGX-UHFFFAOYSA-N 0.000 claims 5
- FBOZXECLQNJBKD-ZDUSSCGKSA-N L-methotrexate Chemical compound C=1N=C2N=C(N)N=C(N)C2=NC=1CN(C)C1=CC=C(C(=O)N[C@@H](CCC(O)=O)C(O)=O)C=C1 FBOZXECLQNJBKD-ZDUSSCGKSA-N 0.000 claims 4
- 229960000485 methotrexate Drugs 0.000 claims 4
- WYWHKKSPHMUBEB-UHFFFAOYSA-N tioguanine Chemical group N1C(N)=NC(=S)C2=C1N=CN2 WYWHKKSPHMUBEB-UHFFFAOYSA-N 0.000 claims 4
- 102100035875 C-C chemokine receptor type 5 Human genes 0.000 claims 3
- 101710149870 C-C chemokine receptor type 5 Proteins 0.000 claims 3
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 3
- 210000003958 hematopoietic stem cell Anatomy 0.000 claims 3
- 150000007523 nucleic acids Chemical group 0.000 claims 3
- 102000018251 Hypoxanthine Phosphoribosyltransferase Human genes 0.000 claims 2
- 108010091358 Hypoxanthine Phosphoribosyltransferase Proteins 0.000 claims 2
- 108091027967 Small hairpin RNA Proteins 0.000 claims 2
- 239000005090 green fluorescent protein Substances 0.000 claims 2
- 239000003112 inhibitor Substances 0.000 claims 2
- 239000004055 small Interfering RNA Substances 0.000 claims 2
- 229960003087 tioguanine Drugs 0.000 claims 2
- 238000011144 upstream manufacturing Methods 0.000 claims 2
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 1
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical group C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims 1
- 108091034151 7SK RNA Proteins 0.000 claims 1
- 108010043121 Green Fluorescent Proteins Proteins 0.000 claims 1
- 102000004144 Green Fluorescent Proteins Human genes 0.000 claims 1
- 102000044159 Ubiquitin Human genes 0.000 claims 1
- 108090000848 Ubiquitin Proteins 0.000 claims 1
- 238000010586 diagram Methods 0.000 claims 1
- 239000002835 hiv fusion inhibitor Substances 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 229920002477 rna polymer Polymers 0.000 claims 1
- 210000000130 stem cell Anatomy 0.000 claims 1
- 239000013598 vector Substances 0.000 claims 1
- 239000013603 viral vector Substances 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2021177252A JP7350361B2 (ja) | 2016-02-19 | 2021-10-29 | 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 |
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 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021177252A Division JP7350361B2 (ja) | 2016-02-19 | 2021-10-29 | 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 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2019509034A JP2019509034A (ja) | 2019-04-04 |
| JP2019509034A5 true JP2019509034A5 (enExample) | 2020-03-19 |
| JP6975465B2 JP6975465B2 (ja) | 2021-12-08 |
Family
ID=59625420
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018543719A Active JP6975465B2 (ja) | 2016-02-19 | 2017-02-17 | 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 |
| JP2021177252A Active JP7350361B2 (ja) | 2016-02-19 | 2021-10-29 | 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 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021177252A Active JP7350361B2 (ja) | 2016-02-19 | 2021-10-29 | 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 |
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 |
|---|---|---|---|---|
| EP4190339B1 (en) | 2017-07-18 | 2025-07-02 | CSL Behring Gene Therapy, Inc. | A modulatable switch for selection of donor modified cells |
| BR112020001059A2 (pt) | 2017-07-18 | 2020-07-14 | Calimmune, Inc. | composições e métodos para o tratamento de beta-hemoglobinopatias |
| KR20210118402A (ko) * | 2018-12-23 | 2021-09-30 | 씨에스엘 베링 엘엘씨 | 위스콧-알드리치(Wiskott-Aldrich) 증후군에 대한 조혈 줄기 세포-유전자 치료요법 |
| JP2022514954A (ja) * | 2018-12-23 | 2022-02-16 | シーエスエル・ベーリング・エルエルシー | キルスイッチを有するドナーｔ細胞 |
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 |
| AU7605796A (en) | 1996-11-04 | 1998-05-29 | 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 |
| DK2284266T3 (da) * | 2002-11-14 | 2014-01-13 | Thermo Fisher Scient Biosciences Inc | sIRNA-MOLEKYLE MOD TP53 |
| US20050266552A1 (en) | 2003-12-05 | 2005-12-01 | Doench John G | 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 |
| WO2015017755A1 (en) | 2013-08-02 | 2015-02-05 | The Regents Of The University Of California | Engineering antiviral t cell immunity through stem cells and chimeric antigen receptors |
| SG10202107602XA (en) * | 2015-11-04 | 2021-08-30 | Univ Pennsylvania | Methods and compositions for gene editing in hematopoietic stem cells |
| BR112020001059A2 (pt) * | 2017-07-18 | 2020-07-14 | Calimmune, Inc. | composições e métodos para o tratamento de beta-hemoglobinopatias |
-
2017
- 2017-02-17 CN CN201780007698.1A patent/CN108495640B/zh active Active
- 2017-02-17 HK HK19100028.5A patent/HK1257668A1/zh unknown
- 2017-02-17 EP EP17753977.2A patent/EP3416660A4/en active Pending
- 2017-02-17 AU AU2017221456A patent/AU2017221456B2/en not_active Ceased
- 2017-02-17 JP JP2018543719A patent/JP6975465B2/ja active Active
- 2017-02-17 US US15/999,854 patent/US11377659B2/en active Active
- 2017-02-17 KR KR1020187021571A patent/KR20180114008A/ko not_active Ceased
- 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
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