JP2018510219A - Ｔａｔ誘導性ｃｒｉｓｐｒ／エンドヌクレアーゼに基づく遺伝子編集 - Google Patents

Ｔａｔ誘導性ｃｒｉｓｐｒ／エンドヌクレアーゼに基づく遺伝子編集 Download PDF

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JP2018510219A
JP2018510219A JP2018500271A JP2018500271A JP2018510219A JP 2018510219 A JP2018510219 A JP 2018510219A JP 2018500271 A JP2018500271 A JP 2018500271A JP 2018500271 A JP2018500271 A JP 2018500271A JP 2018510219 A JP2018510219 A JP 2018510219A
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hiv
sequence
nucleic acid
crispr
acid sequence
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ハリリ，カメル
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テンプル ユニバーシティー オブ ザ コモンウェルス システム オブ ハイヤー エデュケーション
テンプル ユニバーシティー オブ ザ コモンウェルス システム オブ ハイヤー エデュケーション
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JP2018500271A 2015-03-20 2016-03-18 Ｔａｔ誘導性ｃｒｉｓｐｒ／エンドヌクレアーゼに基づく遺伝子編集 Pending JP2018510219A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562136080P 2015-03-20 2015-03-20
US62/136,080 2015-03-20
PCT/US2016/023170 WO2016154016A2 (en) 2015-03-20 2016-03-18 Tat-induced crispr/endonuclease-based gene editing

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JP2018510219A true JP2018510219A (ja) 2018-04-12
JP2018510219A5 JP2018510219A5 (https=) 2019-04-25

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US (3) US20180073019A1 (https=)
EP (1) EP3271021A4 (https=)
JP (1) JP2018510219A (https=)
KR (1) KR20170137114A (https=)
CN (1) CN107949424B (https=)
AU (1) AU2016235472A1 (https=)
BR (1) BR112017019966A2 (https=)
CA (1) CA2980317A1 (https=)
EA (1) EA201792092A1 (https=)
HK (1) HK1254230A1 (https=)
IL (1) IL254480A0 (https=)
MA (1) MA41382A (https=)
MX (1) MX2017011829A (https=)
SG (2) SG11201707458VA (https=)
WO (1) WO2016154016A2 (https=)
ZA (1) ZA201706236B (https=)

Families Citing this family (41)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP6261500B2 (ja) 2011-07-22 2018-01-17 プレジデント アンド フェローズ オブ ハーバード カレッジ ヌクレアーゼ切断特異性の評価および改善
US20150044192A1 (en) 2013-08-09 2015-02-12 President And Fellows Of Harvard College Methods for identifying a target site of a cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
WO2016022363A2 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
EP4491732A3 (en) 2015-10-08 2025-03-26 President and Fellows of Harvard College Multiplexed genome editing
JP7109784B2 (ja) 2015-10-23 2022-08-01 プレジデント アンド フェローズ オブ ハーバード カレッジ 遺伝子編集のための進化したＣａｓ９蛋白質
CA3026332A1 (en) * 2016-06-03 2017-12-14 Temple University - Of The Commonwealth System Of Higher Education Negative feedback regulation of hiv-1 by gene editing strategy
KR102547316B1 (ko) 2016-08-03 2023-06-23 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
US11661590B2 (en) 2016-08-09 2023-05-30 President And Fellows Of Harvard College Programmable CAS9-recombinase fusion proteins and uses thereof
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
EP3526320A1 (en) 2016-10-14 2019-08-21 President and Fellows of Harvard College Aav delivery of nucleobase editors
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
JP2020510038A (ja) 2017-03-09 2020-04-02 プレジデント アンド フェローズ オブ ハーバード カレッジ がんワクチン
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
JP2020510439A (ja) 2017-03-10 2020-04-09 プレジデント アンド フェローズ オブ ハーバード カレッジ シトシンからグアニンへの塩基編集因子
CA3057192A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
US20200140865A1 (en) * 2017-04-17 2020-05-07 Temple University - Of The Commonwealth System Of Higher Education An hiv-1 eradication strategy employing nanoformulated anti-retroviral drugs and gene editing agents
WO2018195402A1 (en) 2017-04-20 2018-10-25 Egenesis, Inc. Methods for generating genetically modified animals
WO2018209320A1 (en) 2017-05-12 2018-11-15 President And Fellows Of Harvard College Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation
WO2019023680A1 (en) 2017-07-28 2019-01-31 President And Fellows Of Harvard College METHODS AND COMPOSITIONS FOR EVOLUTION OF BASIC EDITORS USING PHAGE-ASSISTED CONTINUOUS EVOLUTION (PACE)
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
KR20250107288A (ko) 2017-10-16 2025-07-11 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도
WO2019118949A1 (en) 2017-12-15 2019-06-20 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
CN108559730B (zh) * 2018-01-12 2021-09-24 中国人民解放军第四军医大学 利用CRISPR/Cas9技术构建Hutat2:Fc基因敲入单核细胞的实验方法
EP3797160A1 (en) 2018-05-23 2021-03-31 The Broad Institute Inc. Base editors and uses thereof
US12522807B2 (en) 2018-07-09 2026-01-13 The Broad Institute, Inc. RNA programmable epigenetic RNA modifiers and uses thereof
WO2020081922A1 (en) * 2018-10-18 2020-04-23 University Of Utah Research Foundation Rna-guided transcriptional regulation and methods of using the same for the treatment of back pain
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
AU2019392277A1 (en) * 2018-12-04 2021-06-24 Syngenta Crop Protection Ag Gene silencing via genome editing
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
WO2020160412A1 (en) * 2019-02-01 2020-08-06 Editas Medicine, Inc. Crispr/cas-related methods and compositions targeting virus genomes
JP7657726B2 (ja) 2019-03-19 2025-04-07 ザ ブロード インスティテュート，インコーポレーテッド 編集ヌクレオチド配列を編集するための方法および組成物
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
DE112021002672T5 (de) 2020-05-08 2023-04-13 President And Fellows Of Harvard College Vefahren und zusammensetzungen zum gleichzeitigen editieren beider stränge einer doppelsträngigen nukleotid-zielsequenz
US11643672B2 (en) * 2021-10-01 2023-05-09 The Florida International University Board Of Trusters Inducible CRISPR system expression and applications thereof
CN119372255A (zh) * 2023-07-27 2025-01-28 中国科学院武汉病毒研究所 一种重组病毒载体及其用途

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015031775A1 (en) * 2013-08-29 2015-03-05 Temple University Of The Commonwealth System Of Higher Education Methods and compositions for rna-guided treatment of hiv infection

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
US4501728A (en) 1983-01-06 1985-02-26 Technology Unlimited, Inc. Masking of liposomes from RES recognition
US4837028A (en) 1986-12-24 1989-06-06 Liposome Technology, Inc. Liposomes with enhanced circulation time
US5306631A (en) * 1987-08-21 1994-04-26 University Of Colorado Foundation, Inc. Compositions and method for inhibition of HIV production
US5693508A (en) * 1994-11-08 1997-12-02 Chang; Lung-Ji Retroviral expression vectors containing MoMLV/CMV-IE/HIV-TAR chimeric long terminal repeats
US5853716A (en) * 1995-07-28 1998-12-29 Yale University Genetically engineered chimeric viruses for the treatment of diseases associated with viral transactivators
JP2001510053A (ja) * 1997-07-18 2001-07-31 カイロン コーポレイション レンチウイルスベクター
FR2893631A1 (fr) * 2005-11-21 2007-05-25 Chu Nice Promoteurs inductibles
US9808527B2 (en) * 2006-11-21 2017-11-07 Purdue Research Foundation Methods and compositions for treating HIV infections
JP2012520084A (ja) * 2009-03-13 2012-09-06 レンチゲン コーポレイション 非組み込み型レトロウイルスベクターワクチン
JP6576904B2 (ja) * 2013-04-04 2019-09-18 トラスティーズ・オブ・ダートマス・カレッジ ＨＩＶ−１プロウイルスＤＮＡのｉｎｖｉｖｏ切除のための組成物及び方法
CN104263745B (zh) * 2014-09-16 2017-06-09 中国医学科学院医学实验动物研究所 一种重组免疫缺陷质粒和病毒以及应用

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015031775A1 (en) * 2013-08-29 2015-03-05 Temple University Of The Commonwealth System Of Higher Education Methods and compositions for rna-guided treatment of hiv infection

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
EBINA, H. ET AL., SCIENTIFIC REPORTS, vol. 3:2510, JPN6020007749, 26 August 2013 (2013-08-26), pages 1 - 7, ISSN: 0004223378 *

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