JP6866385B2 - 造血幹細胞における遺伝子編集のための方法および組成物 - Google Patents

造血幹細胞における遺伝子編集のための方法および組成物 Download PDF

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JP6866385B2
JP6866385B2 JP2018543036A JP2018543036A JP6866385B2 JP 6866385 B2 JP6866385 B2 JP 6866385B2 JP 2018543036 A JP2018543036 A JP 2018543036A JP 2018543036 A JP2018543036 A JP 2018543036A JP 6866385 B2 JP6866385 B2 JP 6866385B2
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cells
hspc
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nucleic acid
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JP2018533625A (ja
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ザール ジル
ザール ジル
ミリアム キム
ミリアム キム
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ザ トラスティーズ オブ ザ ユニバーシティ オブ ペンシルバニア
ザ トラスティーズ オブ ザ ユニバーシティ オブ ペンシルバニア
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JP2018543036A 2015-11-04 2016-11-03 造血幹細胞における遺伝子編集のための方法および組成物 Expired - Fee Related JP6866385B2 (ja)

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JP2023097365A JP2023115069A (ja) 2015-11-04 2023-06-14 造血幹細胞における遺伝子編集のための方法および組成物

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Families Citing this family (60)

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Publication number Priority date Publication date Assignee Title
US10323236B2 (en) 2011-07-22 2019-06-18 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
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
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
US10077453B2 (en) 2014-07-30 2018-09-18 President And Fellows Of Harvard College CAS9 proteins including ligand-dependent inteins
US10201606B2 (en) 2014-11-26 2019-02-12 Miltenyi Biotec Gmbh Combination immunotherapy of antigen-recognizing receptors and hematopoietic cells for the treatment of diseases
AU2016337525B2 (en) 2015-10-16 2022-01-20 The Trustees Of Columbia University In The City Of New York Compositions and methods for inhibition of lineage specific antigens
IL310721B2 (en) 2015-10-23 2025-11-01 Harvard College Nucleobase editors and uses thereof
WO2017079400A1 (en) * 2015-11-04 2017-05-11 The Trustees Of The University Of Pennsylvania Methods and compositions for gene editing in hematopoietic stem cells
CN108495640B (zh) * 2016-02-19 2022-11-04 加利福尼亚大学董事会 短发夹rna(shrna734)及其用于阳性选择和消除遗传修饰的细胞的用途
CN110214183A (zh) 2016-08-03 2019-09-06 哈佛大学的校长及成员们 腺苷核碱基编辑器及其用途
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
KR102622411B1 (ko) 2016-10-14 2024-01-10 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
LT3535392T (lt) * 2016-11-02 2024-04-25 Universität Basel Imunologiškai atpažįstami ląstelių paviršiaus variantai, skirti naudoti ląstelių terapijoje
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
RU2019130504A (ru) * 2017-02-28 2021-03-30 Вор Байофарма, Инк. Композиции и способы ингибирования линиеспецифических белков
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
US12390514B2 (en) 2017-03-09 2025-08-19 President And Fellows Of Harvard College Cancer vaccine
US11542496B2 (en) 2017-03-10 2023-01-03 President And Fellows Of Harvard College Cytosine to guanine base editor
KR20240116572A (ko) 2017-03-23 2024-07-29 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제
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
CN111801345A (zh) 2017-07-28 2020-10-20 哈佛大学的校长及成员们 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物
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
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
KR20210045360A (ko) 2018-05-16 2021-04-26 신테고 코포레이션 가이드 rna 설계 및 사용을 위한 방법 및 시스템
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
IL260445B (en) * 2018-07-05 2019-08-29 Lempo Therapeutics Ltd Methods and compositions for modulating myeloperoxidase (mpo) expression
US12522807B2 (en) 2018-07-09 2026-01-13 The Broad Institute, Inc. RNA programmable epigenetic RNA modifiers and uses thereof
CA3110837A1 (en) 2018-08-28 2020-03-05 Vor Biopharma Inc. Genetically engineered hematopoietic stem cells and uses thereof
WO2020061256A1 (en) * 2018-09-19 2020-03-26 FUJIFILM Cellular Dynamics, Inc. Protein l for activation and expansion of chimeric antigen receptor-modified immune cells
US12331320B2 (en) 2018-10-10 2025-06-17 The Research Foundation For The State University Of New York Genome edited cancer cell vaccines
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
WO2020092850A1 (en) * 2018-10-31 2020-05-07 Humanigen, Inc. Materials and methods for treating cancer
CN109852627B (zh) * 2018-12-26 2022-07-05 中国人民解放军军事科学院军事医学研究院 用于CRISPRi系统的质粒、其构建方法及其在使鼠疫菌目的基因定向沉默中的应用
EP3911371A4 (en) * 2019-01-18 2023-04-26 University of Southern California METHODS AND COMPOSITIONS FOR IMPROVING THE SAFETY AND EFFICACY OF CELL THERAPY
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
US20220202900A1 (en) * 2019-02-22 2022-06-30 The Trustees Of The University Of Pennsylvania Compositions and methods for crispr/cas9 knock-out of cd33 in human hematopoietic stem / progenitor cells for allogenic transplantation in patients with relapsed - refractory acute myeloid leukemia
MX2021010559A (es) 2019-03-07 2021-12-15 Univ California Polipéptidos efectores de crispr-cas y métodos de uso de estos.
WO2020191233A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
AU2020338011A1 (en) * 2019-08-28 2022-03-10 Vor Biopharma Inc. Compositions and methods for CLL1 modification
WO2021041977A1 (en) * 2019-08-28 2021-03-04 Vor Biopharma, Inc. Compositions and methods for cd123 modification
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
EP4041297A4 (en) 2019-10-11 2024-02-28 The Trustees of The University of Pennsylvania COMPOSITIONS AND METHODS FOR TARGETING CD13 AND TIM-3 WITH CAR-T CELLS FOR TREATING ACUTE MYELOID LEUKEMIA
KR102923195B1 (ko) 2020-02-28 2026-02-04 현대자동차주식회사 리튬이온 전지 음극 제조방법 및 이에 의해 제조된 리튬이온 전지 음극을 포함하는 리튬이온 전지
IL297761A (en) 2020-05-08 2022-12-01 Broad Inst Inc Methods and compositions for simultaneously editing two helices of a designated double-helix nucleotide sequence
EP3909612A1 (en) * 2020-05-12 2021-11-17 Life Science Inkubator Betriebs GmbH & Co. KG Composition of nanoparticles
JP2024502820A (ja) * 2020-12-31 2024-01-23 ブイオーアール バイオファーマ インコーポレーテッド Cd34遺伝子改変のための組成物及び方法
CN120249215A (zh) * 2021-04-27 2025-07-04 上海驯鹿生物技术有限公司 一种基因编辑的造血干细胞及其与car-t细胞的联合应用
TW202309283A (zh) * 2021-04-28 2023-03-01 日商衛材R&D企管股份有限公司 反義寡核苷酸及其用於治療神經變性障礙之用途
JP2024543369A (ja) * 2021-11-09 2024-11-21 ブイオーアール バイオファーマ インコーポレーテッド Emr2修飾のための化合物及び方法
EP4433512A4 (en) 2021-11-19 2025-12-31 Univ Pennsylvania Genetically modified pan-leukocyte-specific CD45 antigen to facilitate T-lymphocyte therapy
WO2023137335A2 (en) * 2022-01-11 2023-07-20 Actinium Pharmaceuticals, Inc. Methods for treating cd33-positive hematological malignancies
WO2025030010A1 (en) 2023-08-01 2025-02-06 Vor Biopharma Inc. Compositions comprising genetically engineered hematopoietic stem cells and methods of use thereof
WO2025059062A1 (en) * 2023-09-11 2025-03-20 The Trustees Of The University Of Pennsylvania Genetic engineering of human hematopoietic stem/progenitor cells (hspcs) for locus-specific expression of therapeutic proteins

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2008086529A2 (en) * 2007-01-11 2008-07-17 Yale University Compositions and methods for targeted inactivation of hiv cell surface receptors
AU2013329186B2 (en) * 2012-10-10 2019-02-14 Sangamo Therapeutics, Inc. T cell modifying compounds and uses thereof
US9573988B2 (en) * 2013-02-20 2017-02-21 Novartis Ag Effective targeting of primary human leukemia using anti-CD123 chimeric antigen receptor engineered T cells
WO2014191128A1 (en) * 2013-05-29 2014-12-04 Cellectis Methods for engineering t cells for immunotherapy by using rna-guided cas nuclease system
AU2015244039B2 (en) * 2014-04-07 2021-10-21 Novartis Ag Treatment of cancer using anti-CD19 chimeric antigen receptor
AU2016337525B2 (en) * 2015-10-16 2022-01-20 The Trustees Of Columbia University In The City Of New York Compositions and methods for inhibition of lineage specific antigens
WO2017079400A1 (en) * 2015-11-04 2017-05-11 The Trustees Of The University Of Pennsylvania Methods and compositions for gene editing in hematopoietic stem cells

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