JP6886404B2 - 初代造血細胞におけるタンパク質送達 - Google Patents

初代造血細胞におけるタンパク質送達 Download PDF

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JP6886404B2
JP6886404B2 JP2017540255A JP2017540255A JP6886404B2 JP 6886404 B2 JP6886404 B2 JP 6886404B2 JP 2017540255 A JP2017540255 A JP 2017540255A JP 2017540255 A JP2017540255 A JP 2017540255A JP 6886404 B2 JP6886404 B2 JP 6886404B2
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cells
cas9
cell
cxcr4
target
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JP2018503387A5 (enExample
JP2018503387A (ja
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アレクサンダー マーソン
アレクサンダー マーソン
ジェニファー ダウドナ
ジェニファー ダウドナ
ジェフリー ブルーストーン
ジェフリー ブルーストーン
キャスリン シューマン
キャスリン シューマン
スティーヴン リン
スティーヴン リン
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University of California
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
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    • C12N5/0647—Haematopoietic stem cells; Uncommitted or multipotent progenitors
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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JP2017540255A 2015-01-30 2016-01-29 初代造血細胞におけるタンパク質送達 Active JP6886404B2 (ja)

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JP2021082195A JP7114117B2 (ja) 2015-01-30 2021-05-14 初代造血細胞におけるタンパク質送達
JP2022115435A JP7356750B2 (ja) 2015-01-30 2022-07-20 初代造血細胞におけるタンパク質送達

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US201562110187P 2015-01-30 2015-01-30
US62/110,187 2015-01-30
US201562209711P 2015-08-25 2015-08-25
US62/209,711 2015-08-25
PCT/US2016/015836 WO2016123578A1 (en) 2015-01-30 2016-01-29 Protein delivery in primary hematopoietic cells

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JP2018503387A JP2018503387A (ja) 2018-02-08
JP2018503387A5 JP2018503387A5 (enExample) 2019-03-07
JP6886404B2 true JP6886404B2 (ja) 2021-06-16

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JP2017540255A Active JP6886404B2 (ja) 2015-01-30 2016-01-29 初代造血細胞におけるタンパク質送達
JP2021082195A Active JP7114117B2 (ja) 2015-01-30 2021-05-14 初代造血細胞におけるタンパク質送達
JP2022115435A Active JP7356750B2 (ja) 2015-01-30 2022-07-20 初代造血細胞におけるタンパク質送達

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JP2022115435A Active JP7356750B2 (ja) 2015-01-30 2022-07-20 初代造血細胞におけるタンパク質送達

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US (1) US20190388469A1 (enExample)
EP (2) EP3929296A1 (enExample)
JP (3) JP6886404B2 (enExample)
KR (1) KR102605464B1 (enExample)
CN (1) CN107429254B (enExample)
AU (2) AU2016211161C1 (enExample)
ES (1) ES2880473T5 (enExample)
IL (1) IL253498B (enExample)
SG (1) SG11201706059SA (enExample)
WO (1) WO2016123578A1 (enExample)

Families Citing this family (94)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013066438A2 (en) 2011-07-22 2013-05-10 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
EP2841572B1 (en) 2012-04-27 2019-06-19 Duke University Genetic correction of mutated genes
CN105408483A (zh) 2013-03-15 2016-03-16 通用医疗公司 遗传和表观遗传调节蛋白至特定基因组基因座的rna引导的靶向
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
US9163284B2 (en) 2013-08-09 2015-10-20 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
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins 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
US20150166984A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting alpha-antitrypsin point mutations
AU2015298571B2 (en) 2014-07-30 2020-09-03 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
US20200332259A1 (en) * 2014-11-04 2020-10-22 Versiti Blood Research Institute Foundation, Inc. Method to bioengineer designer red blood cells using gene editing and stem cell methodologies
US10676726B2 (en) 2015-02-09 2020-06-09 Duke University Compositions and methods for epigenome editing
CA2986310A1 (en) 2015-05-11 2016-11-17 Editas Medicine, Inc. Optimized crispr/cas9 systems and methods for gene editing in stem cells
US11911415B2 (en) 2015-06-09 2024-02-27 Editas Medicine, Inc. CRISPR/Cas-related methods and compositions for improving transplantation
US11555207B2 (en) 2015-06-17 2023-01-17 The Uab Research Foundation CRISPR/Cas9 complex for introducing a functional polypeptide into cells of blood cell lineage
EP3310932B1 (en) 2015-06-17 2023-08-30 The UAB Research Foundation Crispr/cas9 complex for genomic editing
EP4339287A3 (en) 2015-07-31 2024-05-22 Regents Of The University Of Minnesota Modified cells and methods of therapy
WO2017066497A2 (en) 2015-10-13 2017-04-20 Duke University Genome engineering with type i crispr systems in eukaryotic cells
WO2017070632A2 (en) 2015-10-23 2017-04-27 President And Fellows Of Harvard College Nucleobase editors and uses thereof
US12214054B2 (en) 2015-11-30 2025-02-04 Duke University Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use
EP3402890B1 (en) * 2016-01-12 2025-01-01 SQZ Biotechnologies Company Intracellular delivery of complexes
EP3443081A4 (en) 2016-04-13 2019-10-30 Duke University CRISPR / CAS9-BASED REPRESSORS FOR IN VIVO SHUT-OFF OF GEN-TARGETS AND METHOD OF USE
JP7490211B2 (ja) 2016-07-19 2024-05-27 デューク ユニバーシティ Cpf1に基づくゲノム編集の治療適用
WO2018027078A1 (en) 2016-08-03 2018-02-08 President And Fellows Of Harard College Adenosine nucleobase editors and uses thereof
AU2017308889B2 (en) 2016-08-09 2023-11-09 President And Fellows Of Harvard College Programmable Cas9-recombinase fusion proteins and uses thereof
US12012598B2 (en) 2016-08-12 2024-06-18 Toolgen Incorporated Manipulated immunoregulatory element and immunity altered thereby
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
KR102662249B1 (ko) * 2016-10-14 2024-05-03 더 제너럴 하스피탈 코포레이션 후성적으로 조절되는 부위-특이적 뉴클레아제
KR20240007715A (ko) 2016-10-14 2024-01-16 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
CA3042179A1 (en) * 2016-10-31 2018-05-03 Seattle Children's Hospital (dba Seattle Children's Research Institute) Method for treating autoimmune disease using cd4 t-cells with engineered stabilization of expression of endogenous foxp3 gene
WO2018111947A1 (en) * 2016-12-12 2018-06-21 Integrated Dna Technologies, Inc. Genome editing enhancement
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
WO2018148603A1 (en) * 2017-02-09 2018-08-16 Allen Institute Genetically-tagged stem cell lines and methods of use
KR20190123328A (ko) 2017-03-09 2019-10-31 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 암 백신
WO2018165504A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Suppression of pain by gene editing
CN110914310A (zh) 2017-03-10 2020-03-24 哈佛大学的校长及成员们 胞嘧啶至鸟嘌呤碱基编辑器
IL306092A (en) 2017-03-23 2023-11-01 Harvard College Nucleic base editors that include nucleic acid programmable DNA binding proteins
US12275963B2 (en) 2017-05-08 2025-04-15 Toolgen Incorporated Artificially manipulated immune cell
US11560566B2 (en) 2017-05-12 2023-01-24 President And Fellows Of Harvard College Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation
MY200418A (en) 2017-06-15 2023-12-25 Univ California Targeted non-viral dna insertions
EP3652312A1 (en) 2017-07-14 2020-05-20 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
EP3658573A1 (en) 2017-07-28 2020-06-03 President and Fellows of Harvard College Methods and compositions for evolving base 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
CN111225975A (zh) * 2017-09-11 2020-06-02 加利福尼亚大学董事会 抗体介导的cas9向哺乳动物细胞的递送
CA3082251A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. Uses of adenosine base editors
SG11202003798TA (en) 2017-10-27 2020-05-28 Univ California Targeted replacement of endogenous t cell receptors
WO2019106522A1 (en) * 2017-11-28 2019-06-06 Novartis Ag Pooled crispr/cas9 screening in primary cells using guide swap technology
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
US12509492B2 (en) 2018-01-19 2025-12-30 Duke University Genome engineering with CRISPR-Cas systems in eukaryotes
EP3765094A4 (en) 2018-03-15 2021-12-22 KSQ Therapeutics, Inc. GENE REGULATORY COMPOSITIONS AND PROCEDURES FOR IMPROVED IMMUNOTHERAPY
SG11202007878UA (en) * 2018-04-27 2020-09-29 Seattle Childrens Hospital Dba Seattle Childrens Res Inst Expression of foxp3 in edited cd34+ cells
JP7575949B2 (ja) * 2018-04-27 2024-10-31 シアトル チルドレンズ ホスピタル ディー/ビー/エイ シアトル チルドレンズ リサーチ インスティテュート 遺伝子編集t細胞におけるヒトfoxp3の発現
CN112654710A (zh) 2018-05-16 2021-04-13 辛瑟高公司 用于指导rna设计和使用的方法和系统
WO2019226953A1 (en) 2018-05-23 2019-11-28 The Broad Institute, Inc. Base editors and uses thereof
US12473533B2 (en) 2018-07-09 2025-11-18 The Regents Of The University Of California Gene targets for T-cell-based immunotherapy
EP3820495A4 (en) 2018-07-09 2022-07-20 The Broad Institute Inc. RNA-PROGRAMMABLE EPIGENETIC RNA MODIFIERS AND THEIR USES
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
CN110527695B (zh) * 2019-03-07 2020-06-16 山东舜丰生物科技有限公司 一种用于基因定点突变的核酸构建物
WO2020191241A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
WO2020198151A1 (en) * 2019-03-22 2020-10-01 Spotlight Therapeutics Targeted active gene editing agent and methods of use
CN114040775A (zh) * 2019-03-22 2022-02-11 光点治疗公司 靶向活性基因编辑剂和使用方法
TWI887234B (zh) 2019-04-14 2025-06-21 美國公爵大學 治療杜興氏(duchenne)肌肉失養症之大型突變熱點之aav載體介導之刪除
WO2020214619A1 (en) * 2019-04-15 2020-10-22 Emendobio Inc. Crispr compositions and methods for promoting gene editing of gata2
WO2020214842A1 (en) 2019-04-17 2020-10-22 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
WO2021062201A1 (en) * 2019-09-25 2021-04-01 Spotlight Therapeutics Compositions and methods for nucleoprotein targeting and expression
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
CA3177481A1 (en) 2020-05-08 2021-11-11 David R. Liu Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
US20230407337A1 (en) * 2020-12-08 2023-12-21 The Regents if the University of California Crispr ribonucleoprotein complex genome editing of human innate immune cells
AU2022318664A1 (en) 2021-07-30 2024-02-29 Tune Therapeutics, Inc. Compositions and methods for modulating expression of methyl-cpg binding protein 2 (mecp2)
US20240254483A1 (en) 2021-07-30 2024-08-01 Tune Therapeutics, Inc. Compositions and methods for modulating expression of frataxin (fxn)
MX2024003887A (es) 2021-10-14 2024-07-09 Arsenal Biosciences Inc Células inmunitarias que tienen arnch coespresados y sistemas de compuerta lógica.
KR20230107750A (ko) * 2022-01-07 2023-07-18 주식회사 툴젠 유전자 편집 과정에서 발생 가능한 오프 타겟을 예측하는 방법
US20250134999A1 (en) 2022-01-14 2025-05-01 Tune Therapeutics, Inc. Compositions, systems, and methods for programming t cell phenotypes through targeted gene activation
EP4463549A2 (en) 2022-01-14 2024-11-20 Tune Therapeutics, Inc. Compositions, systems, and methods for programming t cell phenotypes through targeted gene repression
CA3260237A1 (en) 2022-06-24 2023-12-28 Tune Therapeutics, Inc. Compositions, systems and methods for reducing low-density lipoprotein by targeted gene repression
EP4554967A2 (en) 2022-07-12 2025-05-21 Tune Therapeutics, Inc. Compositions, systems, and methods for targeted transcriptional activation
WO2024040254A2 (en) 2022-08-19 2024-02-22 Tune Therapeutics, Inc. Compositions, systems, and methods for regulation of hepatitis b virus through targeted gene repression
JP2025531268A (ja) 2022-09-19 2025-09-19 チューン セラピューティクス インコーポレイテッド T細胞機能を調節するための組成物、システム、および方法
WO2024127369A1 (en) * 2022-12-16 2024-06-20 LifeEDIT Therapeutics, Inc. Guide rnas that target foxp3 gene and methods of use
WO2024163678A2 (en) 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Fusion proteins and systems for targeted activation of frataxin (fxn) and related methods
WO2024163683A2 (en) 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Systems, compositions, and methods for modulating expression of methyl-cpg binding protein 2 (mecp2) and x-inactive specific transcript (xist)
CN116496992B (zh) * 2023-04-24 2023-12-01 江苏省家禽科学研究所 一种鸡胚成肌永生化细胞及其构建方法和应用
CN121909284A (zh) 2023-07-31 2026-04-21 图恩疗法股份有限公司 用于调节il-2基因表达的组合物和方法
EP4754248A1 (en) 2023-07-31 2026-06-10 Tune Therapeutics, Inc. Compositions and methods for multiplexed activation and repression of t cell gene expression
WO2025038494A1 (en) 2023-08-11 2025-02-20 Tune Therapeutics, Inc. Compositions, systems, and methods for lymphoid cell differentiation using targeted gene activation
WO2025059073A1 (en) 2023-09-11 2025-03-20 Tune Therapeutics, Inc. Epigenetic editing methods and systems for differentiating stem cells
CN117448330B (zh) * 2023-10-25 2025-01-21 山东省农业科学院家禽研究所(山东省无特定病原鸡研究中心) Acsl4基因缺失的mdck纯合子细胞系的构建方法和应用
WO2025260068A1 (en) 2024-06-14 2025-12-18 Tune Therapeutics, Inc. Lipid nanoparticle formulation for delivery of nucleic acids to cells
WO2026015647A1 (en) 2024-07-09 2026-01-15 Tune Therapeutics, Inc. Compositions, systems, and methods for cell differentiation using targeted gene activation of dll4 and/or vcam1
WO2026064753A1 (en) 2024-09-23 2026-03-26 Tune Therapeutics, Inc. Dna methyltransferase-like protein (dnmt3l) or dna methyltransferase 3a (dnmt3a) repressor systems for epigenetic editing
WO2026102058A1 (en) 2024-11-06 2026-05-15 Tune Therapeutics, Inc. Methods for treating chronic hepatitis b infection and related formulations
WO2026161895A1 (en) 2025-01-27 2026-07-30 Tune Therapeutics, Inc. Compositions, systems, and methods for epigenetic regulation of lipoprotein a (lpa) gene expression

Family Cites Families (36)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US702996A (en) 1901-12-05 1902-06-24 Wladyslaw Pruszkowski Alloy.
US6773669B1 (en) 1995-03-10 2004-08-10 Maxcyte, Inc. Flow electroporation chamber and method
US6090617A (en) 1996-12-05 2000-07-18 Entremed, Inc. Flow electroporation chamber with electrodes having a crystalline metal nitride coating
AU2472400A (en) 1998-10-20 2000-05-08 City Of Hope CD20-specific redirected T cells and their use in cellular immunotherapy of CD20+ malignancies
US7070995B2 (en) 2001-04-11 2006-07-04 City Of Hope CE7-specific redirected immune cells
ES2464532T3 (es) 2001-04-19 2014-06-03 The Scripps Research Institute Métodos y composiciones para la producción de pares ortogonales de ARNt-aminoacil-ARNt sintetasa
JP4070611B2 (ja) 2001-04-23 2008-04-02 アマクサ アーゲー 電気穿孔法用の緩衝溶液およびその使用を含む方法
DK2574662T3 (da) 2001-08-22 2021-09-20 Maxcyte Inc Fremgangsmåde til elektroporation af biologiske prøver
US7446190B2 (en) 2002-05-28 2008-11-04 Sloan-Kettering Institute For Cancer Research Nucleic acids encoding chimeric T cell receptors
US20050129671A1 (en) 2003-03-11 2005-06-16 City Of Hope Mammalian antigen-presenting T cells and bi-specific T cells
DE502004009820D1 (de) 2004-03-15 2009-09-10 Lonza Cologne Ag Behältnis und Vorrichtung zur Erzeugung von elektrischen Feldern in einzelnen Reaktionsräumen
WO2005113820A2 (en) 2004-05-12 2005-12-01 Maxcyte, Inc. Methods and devices related to a regulated flow electroporation chamber
RU2006143560A (ru) 2004-06-12 2008-07-20 Диджитал Био Технолоджи Ко Устройство для электропорации с удлиненным полым элементом
ES2465467T3 (es) 2004-06-14 2014-06-05 Lonza Cologne Ag Procedimiento y disposición de circuito para el tratamiento de material biológico
JP2008521437A (ja) 2004-11-30 2008-06-26 マックスサイト インコーポレーティッド コンピュータ化されたエレクトロポレーション
JP5043672B2 (ja) 2005-10-28 2012-10-10 田辺三菱製薬株式会社 新規細胞膜透過ペプチド
JP5779090B2 (ja) 2008-04-09 2015-09-16 マックスサイト インコーポレーティッド 新規に単離された細胞の治療組成物の操作および送達
WO2009147368A1 (en) 2008-06-04 2009-12-10 Medical Research Council Peptides
EP2310500B1 (en) 2008-07-18 2015-03-11 Maxcyte, Inc. Methods for optimizing electroporation
CA2735456C (en) 2008-08-26 2021-11-16 City Of Hope Method and compositions for enhanced anti-tumor effector functioning of t cells
US8389481B2 (en) 2008-11-12 2013-03-05 University Of Medicine And Dentistry Of New Jersey Glutamate-enhanced cell-penetrating peptides and methods of using same
WO2010121130A2 (en) 2009-04-16 2010-10-21 President And Fellows Of Harvard College Molecular delivery with nanowires
AU2010250452B2 (en) 2009-05-20 2014-06-05 Toray Industries, Inc. Cell membrane-permeable peptides
US8372951B2 (en) 2010-05-14 2013-02-12 National Tsing Hua University Cell penetrating peptides for intracellular delivery
WO2012129514A1 (en) 2011-03-23 2012-09-27 Fred Hutchinson Cancer Research Center Method and compositions for cellular immunotherapy
JP6053688B2 (ja) 2011-10-07 2016-12-27 国立大学法人三重大学 キメラ抗原受容体
WO2013059343A1 (en) * 2011-10-17 2013-04-25 Massachusetts Institute Of Technology Intracellular delivery
JP6267644B2 (ja) 2011-10-20 2018-01-24 アメリカ合衆国 抗cd22キメラ抗原受容体
CA2868121C (en) 2012-03-23 2021-06-01 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Anti-mesothelin chimeric antigen receptors
ES2728782T3 (es) 2012-05-25 2019-10-28 Univ California Métodos y composiciones para la modificación de ADN objetivo dirigida por ARN y para la modulación de la transcripción dirigida por ARN
US8614194B1 (en) 2012-07-25 2013-12-24 Kaohsiung Medical University Anionic cell penetrating peptide and its use for intracellular delivery
WO2014031173A1 (en) 2012-08-22 2014-02-27 President And Fellows Of Harvard College Multiwell plates comprising nanowires
EP2931899A1 (en) * 2012-12-12 2015-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
IL298125A (en) 2013-02-26 2023-01-01 Memorial Sloan Kettering Cancer Center Preparations and methods for immunotherapy
JP6608807B2 (ja) * 2013-05-29 2019-11-20 セレクティス Rnaガイドcasヌクレアーゼ系を用いることによって免疫療法のためにt細胞を操作するための方法
CN106536721B (zh) 2014-08-06 2020-12-04 车医科学大学校产学协力团 核酸酶介导的编辑编码hla的基因所产生的免疫相容性细胞

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