JP2016514479A - HIV−1プロウイルスDNAのinvivo切除のための組成物及び方法 - Google Patents
HIV−1プロウイルスDNAのinvivo切除のための組成物及び方法 Download PDFInfo
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Abstract
Description
EVDGVDEVAKKKSKK (配列番号26) (Schreiberら(1992) EMBO J. 11:3263-3269)又はステロイドホルモン受容体(ヒト)グルココルチコイドの配列RKCLQAGMNLEARKTKK (配列番号27) (Cadepondら(1992) Exp. Cell Res. 201:99-108)等の二分核局在配列を用いることも可能である。ある実施態様において、NLSはcasタンパク質のN末端に局在する。他の実施態様において、NLSはcasタンパク質のC末端に局在する。
HIV-1プロウイルスの長鎖末端反復領域内に位置する5種類の特定の領域の一つに特異的に結合するガイドRNA分子をコードするプラスミドDNAを作製する(図1)。同様に、cas9ヌクレアーゼをコードするプラスミドを作製する。両方のプラスミドをヒト細胞に送達し、そこでcas9及びガイドRNA遺伝子座が転写され、cas9転写産物は翻訳される。続いて、ガイドRNA部分がcas9ヌクレアーゼに結合し、ハイブリッド複合体を形成する。核局在配列を加えているので、このハイブリッド複合体は核内に入り、核においてガイドRNA部分がHIV-1プロウイルスDNA内の相補配列に結合する。ヌクレアーゼがプロウイルスDNAを切り、通常修復不可能な二重らせんDNA切片をもたらす。図2を参照。この方法を用いることにより、HIV-1プロウイルスDNAを感染細胞のゲノムから切除することができる。
J-Lat細胞株(ヒトT細胞がん株)を用いて、ガイドRNA:hCas9複合体によるHIV-1長鎖末端反復(LTR)の切断がこれらの細胞の転写活性を変化させるか否かを決定した。フローサイトメトリーを用いてトランスフェクション22時間後にLTRプロモーター由来の緑色蛍光タンパク質(GFP)を生産する細胞を定量した。フローサイトメトリー解析により、GFPを発現する細胞の割合が得られ、またGFP+細胞の平均蛍光強度(MFI)を定量することにより細胞群内の発現の強度を定量することもできる。フローサイトメトリーにより、転写を減少させるLTR領域内の変異と、転写を完全に消失させるLTR領域内の変異とを識別することができる。
CRISPR/Cas法のHIV-1 LTR内での切断及び転写の改変の有効性を決定するために、Jurkat細胞を3種類の異なるプラスミドでトランスフェクションした。これらのプラスミドには、様々なガイドRNA、ヒト化Cas9プラスミド、及び無傷のHIV-1 LTR及び5'配列がGFPの発現を促進する第3の(レポーター)プラスミドが含まれる。
Claims (11)
- 真核細胞における標的のヒト免疫不全ウイルス1(HIV-1)のDNA配列の機能又は存在を阻害する方法であって、標的HIV-1 DNA配列を保有する真核細胞を、
(a) 一種以上のガイドRNA、又は前記一種以上のガイドRNAをコードする核酸、及び
(b) 規則的な間隔をもってクラスター化された短鎖反復回文配列関連(Clustered Regularly Interspaced Short Palindromic Repeats-Associated:cas)タンパク質、又は前記casタンパク質をコードする核酸
と接触させる工程を含み、
前記ガイドRNAが前記標的HIV-1 DNA配列とハイブリダイズし、これにより前記標的HIV-1 DNA配列の機能又は存在を阻害するものである、方法。 - 前記標的HIV-1 DNA配列が配列番号2、配列番号3、配列番号4、配列番号5、配列番号6、配列番号7、配列番号8、配列番号9及び配列番号10から成る群から選択される、請求項1に記載の方法。
- 前記casタンパク質がcas9である、請求項1に記載の方法。
- 前記casタンパク質がヒト細胞での発現用にコドン最適化されている、請求項1に記載の方法。
- 前記casタンパク質が核局在配列をさらに含む、請求項1に記載の方法。
- (a) 一種以上のガイドRNA、又は前記一種以上のガイドRNAをコードする核酸であって、前記ガイドRNAが標的のヒト免疫不全ウイルス1 (HIV-1)のDNA配列とハイブリダイズするもの;及び
(b) 規則的な間隔をもってクラスター化された短鎖反復回文配列関連(Clustered Regularly Interspaced Short Palindromic Repeats-Associated:cas)タンパク質;又は前記タンパク質をコードする核酸
を含むキット。 - 標的HIV-1 DNA配列が、配列番号2、配列番号3、配列番号4、配列番号5、配列番号6、配列番号7、配列番号8、配列番号9及び配列番号10から成る群から選択される、請求項6に記載のキット。
- 前記casタンパク質がcas9である、請求項6に記載のキット。
- 前記casタンパク質がヒト細胞での発現用にコドン最適化されている、請求項6に記載のキット。
- 前記casタンパク質が核局在配列をさらに含む、請求項6に記載のキット。
- 一種以上のガイドRNAをコードする核酸を含み、前記ガイドRNAが配列番号2、配列番号3、配列番号4、配列番号5、配列番号6、配列番号7、配列番号8、配列番号9及び配列番号10から成る群から選択される標的のヒト免疫不全ウイルス1のDNA配列とハイブリダイズする、ベクター。
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Publication number | Priority date | Publication date | Assignee | Title |
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JP2016534125A (ja) * | 2013-08-29 | 2016-11-04 | テンプル ユニヴァーシティ オブ ザ コモンウェルス システム オブ ハイヤー エデュケイション | Hiv感染のrnaガイド処置のための方法および組成物 |
Families Citing this family (63)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
CA2853829C (en) | 2011-07-22 | 2023-09-26 | President And Fellows Of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
EP3434776A1 (en) | 2012-12-12 | 2019-01-30 | The Broad Institute, Inc. | Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof |
EP2931897B1 (en) | 2012-12-12 | 2017-11-01 | The Broad Institute, Inc. | Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications |
EP2946015B1 (en) | 2013-01-16 | 2021-05-26 | Emory University | Cas9-nucleic acid complexes and uses related thereto |
EP2981612B1 (en) * | 2013-04-04 | 2019-07-03 | Trustees of Dartmouth College | Compositions and methods for in vivo excision of hiv-1 proviral dna |
CN107995927B (zh) | 2013-06-17 | 2021-07-30 | 布罗德研究所有限公司 | 用于肝靶向和治疗的crispr-cas系统、载体和组合物的递送与用途 |
EP3725885A1 (en) | 2013-06-17 | 2020-10-21 | The Broad Institute, Inc. | Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof |
AU2014281027A1 (en) | 2013-06-17 | 2016-01-28 | Massachusetts Institute Of Technology | Optimized CRISPR-Cas double nickase systems, methods and compositions for sequence manipulation |
CN106062197A (zh) | 2013-06-17 | 2016-10-26 | 布罗德研究所有限公司 | 用于序列操纵的串联指导系统、方法和组合物的递送、工程化和优化 |
KR20160056869A (ko) | 2013-06-17 | 2016-05-20 | 더 브로드 인스티튜트, 인코퍼레이티드 | 바이러스 구성성분을 사용하여 장애 및 질환을 표적화하기 위한 crispr-cas 시스템 및 조성물의 전달, 용도 및 치료 적용 |
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 |
US9388430B2 (en) | 2013-09-06 | 2016-07-12 | President And Fellows Of Harvard College | Cas9-recombinase fusion proteins and uses thereof |
US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
US9340800B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | Extended DNA-sensing GRNAS |
KR102380245B1 (ko) | 2013-11-07 | 2022-03-30 | 에디타스 메디신, 인코포레이티드 | 지배적인 gRNA를 이용하는 CRISPR-관련 방법 및 조성물 |
MX2016007325A (es) | 2013-12-12 | 2017-07-19 | Broad Inst Inc | Composiciones y metodos de uso de sistemas crispr-cas en desordenes debidos a repeticion de nucleotidos. |
US20150166982A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting pi3k point mutations |
EP3080271B1 (en) | 2013-12-12 | 2020-02-12 | The Broad Institute, Inc. | Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems |
WO2015089364A1 (en) | 2013-12-12 | 2015-06-18 | The Broad Institute Inc. | Crystal structure of a crispr-cas system, and uses thereof |
CN105899658B (zh) * | 2013-12-12 | 2020-02-18 | 布罗德研究所有限公司 | 针对hbv和病毒性疾病以及障碍的crispr-cas系统和组合物的递送、用途和治疗应用 |
CA2932478A1 (en) | 2013-12-12 | 2015-06-18 | Massachusetts Institute Of Technology | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for genome editing |
WO2015184268A1 (en) | 2014-05-30 | 2015-12-03 | The Board Of Trustees Of The Leland Stanford Junior University | Compositions and methods of delivering treatments for latent viral infections |
US10077453B2 (en) | 2014-07-30 | 2018-09-18 | President And Fellows Of Harvard College | CAS9 proteins including ligand-dependent inteins |
WO2016057061A2 (en) * | 2014-10-10 | 2016-04-14 | Massachusetts Eye And Ear Infirmary | Efficient delivery of therapeutic molecules in vitro and in vivo |
WO2016086177A2 (en) * | 2014-11-25 | 2016-06-02 | Drexel University | Compositions and methods for hiv quasi-species excision from hiv-1-infected patients |
US10900034B2 (en) | 2014-12-03 | 2021-01-26 | Agilent Technologies, Inc. | Guide RNA with chemical modifications |
EP3985115A1 (en) | 2014-12-12 | 2022-04-20 | The Broad Institute, Inc. | Protected guide rnas (pgrnas) |
MA41382A (fr) * | 2015-03-20 | 2017-11-28 | Univ Temple | Édition génique basée sur le système crispr/endonucléase à induction par tat |
EP3280803B1 (en) | 2015-04-06 | 2021-05-26 | The Board of Trustees of the Leland Stanford Junior University | Chemically modified guide rnas for crispr/cas-mediated gene regulation |
CA2985650A1 (en) * | 2015-05-13 | 2016-11-17 | Seattle Children's Hospital (dba Seattle Children's Research Institute) | Enhancing endonuclease based gene editing in primary cells |
CA3000182A1 (en) * | 2015-05-29 | 2016-12-08 | Agenovir Corporation | Methods and compositions for treating cells for transplant |
US10117911B2 (en) | 2015-05-29 | 2018-11-06 | Agenovir Corporation | Compositions and methods to treat herpes simplex virus infections |
US20160346360A1 (en) * | 2015-05-29 | 2016-12-01 | Agenovir Corporation | Compositions and methods for cell targeted hpv treatment |
PL3302709T3 (pl) * | 2015-06-01 | 2021-12-06 | Temple University - Of The Commonwealth System Of Higher Education | Sposoby i kompozycje do leczenia zakażenia HIV przy użyciu RNA naprowadzającego |
WO2016205759A1 (en) | 2015-06-18 | 2016-12-22 | The Broad Institute Inc. | Engineering and optimization of systems, methods, enzymes and guide scaffolds of cas9 orthologs and variants for sequence manipulation |
TWI813532B (zh) | 2015-06-18 | 2023-09-01 | 美商博得學院股份有限公司 | 降低脱靶效應的crispr酶突變 |
JP2018527943A (ja) * | 2015-09-28 | 2018-09-27 | 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 | Rna誘導性の、hiv感染の処置のための、方法および組成物 |
WO2017066588A2 (en) * | 2015-10-16 | 2017-04-20 | Temple University - Of The Commonwealth System Of Higher Education | Methods and compositions utilizing cpf1 for rna-guided gene editing |
EP4269577A3 (en) | 2015-10-23 | 2024-01-17 | President and Fellows of Harvard College | Nucleobase editors and uses thereof |
WO2017132112A1 (en) * | 2016-01-25 | 2017-08-03 | Excision Biotherapeutics | Methods and compositions for rna-guided treatment of hiv infection |
EP3219799A1 (en) | 2016-03-17 | 2017-09-20 | IMBA-Institut für Molekulare Biotechnologie GmbH | Conditional crispr sgrna expression |
US20190225956A1 (en) * | 2016-05-10 | 2019-07-25 | United States Government As Represented By The Department Of Veterans Affairs | Lentiviral delivery of crispr/cas constructs that cleave genes essential for hiv-1 infection and replication |
US10767175B2 (en) | 2016-06-08 | 2020-09-08 | Agilent Technologies, Inc. | High specificity genome editing using chemically modified guide RNAs |
SG11201900907YA (en) | 2016-08-03 | 2019-02-27 | Harvard College | Adenosine nucleobase editors and uses thereof |
WO2018031683A1 (en) | 2016-08-09 | 2018-02-15 | 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 |
CN110214180A (zh) | 2016-10-14 | 2019-09-06 | 哈佛大学的校长及成员们 | 核碱基编辑器的aav递送 |
US10745677B2 (en) | 2016-12-23 | 2020-08-18 | President And Fellows Of Harvard College | Editing of CCR5 receptor gene to protect against HIV infection |
TW201839136A (zh) | 2017-02-06 | 2018-11-01 | 瑞士商諾華公司 | 治療血色素異常症之組合物及方法 |
EP3592853A1 (en) | 2017-03-09 | 2020-01-15 | President and Fellows of Harvard College | Suppression of pain by gene editing |
EP3592777A1 (en) | 2017-03-10 | 2020-01-15 | President and Fellows of Harvard College | Cytosine to guanine base editor |
CN110914426A (zh) | 2017-03-23 | 2020-03-24 | 哈佛大学的校长及成员们 | 包含核酸可编程dna结合蛋白的核碱基编辑器 |
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) |
US11319532B2 (en) | 2017-08-30 | 2022-05-03 | President And Fellows Of Harvard College | High efficiency base editors comprising Gam |
JP2021500036A (ja) | 2017-10-16 | 2021-01-07 | ザ ブロード インスティテュート, インコーポレーテッドThe Broad Institute, Inc. | アデノシン塩基編集因子の使用 |
BR112021018607A2 (pt) | 2019-03-19 | 2021-11-23 | Massachusetts Inst Technology | Métodos e composições para editar sequências de nucleotídeos |
CN116096873A (zh) | 2020-05-08 | 2023-05-09 | 布罗德研究所股份有限公司 | 同时编辑靶标双链核苷酸序列的两条链的方法和组合物 |
MX2023000661A (es) * | 2020-07-13 | 2023-07-03 | Alexandra L Howell | Métodos y composiciones para la eficiencia y especificidad de arn guía de crispr/cas9 frente a aislados de vih-1 genéticamente diferentes. |
AU2022343300A1 (en) | 2021-09-10 | 2024-04-18 | Agilent Technologies, Inc. | Guide rnas with chemical modification for prime editing |
WO2024064800A2 (en) * | 2022-09-21 | 2024-03-28 | Board Of Regents Of The University Of Nebraska | Lipid nanoparticle formulations and methods of use thereof |
JP7388586B1 (ja) | 2023-03-20 | 2023-11-29 | 三菱電機ビルソリューションズ株式会社 | エレベーターシステム |
Citations (1)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2014093622A2 (en) * | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications |
Family Cites Families (17)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
US5866701A (en) | 1988-09-20 | 1999-02-02 | The Board Of Regents For Northern Illinois University Of Dekalb | HIV targeted hairpin ribozymes |
ATE157012T1 (de) | 1989-11-03 | 1997-09-15 | Univ Vanderbilt | Verfahren zur in vivo-verabreichung von funktionsfähigen fremden genen |
US5670488A (en) | 1992-12-03 | 1997-09-23 | Genzyme Corporation | Adenovirus vector for gene therapy |
US5837484A (en) | 1993-11-09 | 1998-11-17 | Medical College Of Ohio | Stable cell lines capable of expressing the adeno-associated virus replication gene |
US5658785A (en) | 1994-06-06 | 1997-08-19 | Children's Hospital, Inc. | Adeno-associated virus materials and methods |
US5559099A (en) | 1994-09-08 | 1996-09-24 | Genvec, Inc. | Penton base protein and methods of using same |
WO1996017947A1 (en) | 1994-12-06 | 1996-06-13 | Targeted Genetics Corporation | Packaging cell lines for generation of high titers of recombinant aav vectors |
US5770442A (en) | 1995-02-21 | 1998-06-23 | Cornell Research Foundation, Inc. | Chimeric adenoviral fiber protein and methods of using same |
ES2150832B1 (es) | 1996-06-12 | 2001-06-16 | Fichtel & Sachs Ag | Dispositivo de maniobra para la maniobra, en particular maniobra neumatica, de un embrague de friccion. |
WO1998027207A1 (en) | 1996-12-18 | 1998-06-25 | Targeted Genetics Corporation | Recombinase-activatable aav packaging cassettes for use in the production of aav vectors |
US5922315A (en) | 1997-01-24 | 1999-07-13 | Genetic Therapy, Inc. | Adenoviruses having altered hexon proteins |
EP1080218A1 (en) | 1998-05-27 | 2001-03-07 | University of Florida | Method of preparing recombinant adeno-associated virus compositions by using an iodixanol gradient |
EP1942192A1 (en) * | 2007-01-08 | 2008-07-09 | Heinrich-Pette-Institut für experimentelle Virologie und Immunologie | Use of a tailored recombinase for the treatment of retroviral infections |
US20100076057A1 (en) | 2008-09-23 | 2010-03-25 | Northwestern University | TARGET DNA INTERFERENCE WITH crRNA |
US9163289B2 (en) | 2010-08-27 | 2015-10-20 | Hanwha Techwin Co., Ltd. | Kit for detecting HIV-1 and method for detecting HIV-1 using the same |
EP2981612B1 (en) * | 2013-04-04 | 2019-07-03 | Trustees of Dartmouth College | Compositions and methods for in vivo excision of hiv-1 proviral dna |
-
2014
- 2014-03-25 EP EP14779556.1A patent/EP2981612B1/en active Active
- 2014-03-25 CA CA2908253A patent/CA2908253C/en active Active
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Patent Citations (1)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2014093622A2 (en) * | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications |
Non-Patent Citations (5)
Title |
---|
COLD SPRING HARBOR PERSPECTIVES IN MEDICINE, 2012, VOL.4, A006916, JPN6018006614, ISSN: 0004031802 * |
NATURE BIOTECHNOLOGY, MARCH 2013, VOL.31, P.230-232, JPN6018006609, ISSN: 0004031801 * |
NUCLEIC ACIDS RESEARCH, 2005, VOL.33, P.235-243, JPN6018006605, ISSN: 0004031799 * |
RNA GUIDED HUMAN GENE AND GENOME ENGONEERING AND RADICAL LIFE EXTENSION, DISEASE PREVENTION AND CURE, JPN6018006611, ISSN: 0003745358 * |
SCIENCE, 15 FEBRUARY 2013, VOL.339, P.823-826, JPN6018006607, ISSN: 0004031800 * |
Cited By (4)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
JP2016534125A (ja) * | 2013-08-29 | 2016-11-04 | テンプル ユニヴァーシティ オブ ザ コモンウェルス システム オブ ハイヤー エデュケイション | Hiv感染のrnaガイド処置のための方法および組成物 |
US11285193B2 (en) | 2013-08-29 | 2022-03-29 | Temple University—Of the Commonwealth System of Higher Education | Methods and compositions for RNA-guided treatment of HIV infection |
US11291710B2 (en) | 2013-08-29 | 2022-04-05 | Temple University—Of the Commonwealth System of Higher Education | Methods and compositions for RNA-guided treatment of HIV infection |
US11298411B2 (en) | 2013-08-29 | 2022-04-12 | Temple University—Of the Commonwealth System of Higher Education | Methods and compositions for RNA-guided treatment of HIV infection |
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US20160040165A1 (en) | 2016-02-11 |
ES2747833T3 (es) | 2020-03-11 |
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JP6576904B2 (ja) | 2019-09-18 |
JP2019198329A (ja) | 2019-11-21 |
EP2981612B1 (en) | 2019-07-03 |
JP2022001051A (ja) | 2022-01-06 |
JP7206338B2 (ja) | 2023-01-17 |
US20220154188A1 (en) | 2022-05-19 |
WO2014165349A1 (en) | 2014-10-09 |
CA2908253A1 (en) | 2014-10-09 |
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US11274305B2 (en) | 2022-03-15 |
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